WANT TO LEARN MORE?
Attending a TAKHZYRO+YOU event is a great way to learn and discover more about TAKHZYRO. You'll hear from healthcare professionals as well as people taking TAKHZYRO.
Find an in-person, virtual, or on-demand event below.
Connect with the HAE community at an educational event.
To find programs in your area, enter your ZIP code and preferred location below. Please make sure to register for an event you plan to attend. You can also check out past webinar recordings here.
If you can't attend an in-person or virtual event, check out past webinar recordings below.
Filter
- 10 mi
- 25 mi
- 50 mi
- 100 mi
- English
- Spanish
- All
View Transcript
KIM: TAKHZYRO® (lanadelumab-flyo) is a prescription medicine used to prevent attacks of hereditary angioedema (HAE) in patients 2 years of age and older. TAKHZYRO may cause serious side effects, including allergic reactions. Continue watching until the end of this video for additional Important Safety Information.
KIM: Hi! I’m Kim, and I’m a physician assistant that works with the HAE community. You’ve made the decision to start preventive treatment with TAKHZYRO for your hereditary angioedema, or HAE, attacks. TAKHZYRO is an important part of the HAE management plan you created with your HAE specialist to help prevent HAE attacks. Today, I’m going to show you what your first few months of treatment with TAKHZYRO may look like. But...I won’t be doing it alone.
LINDA: Hello! I'm Linda, and I started treatment with TAKHZYRO within a month of its approval. Whether you've been on preventive treatment before or TAKHZYRO is your first experience with one, we know this is a big decision on your HAE journey. I remember when I first started treatment, it felt overwhelming at times. I had never tried preventive treatment before. It was hard to picture how it would all play out, but in the end, I'm glad I stuck with it. Throughout this video, I'll be sharing some of my experiences and even a little advice in the hopes that they will help you as you begin treatment with TAKHZYRO.
LINDA: If you’re like me, you and your doctor chose TAKHZYRO because you wanted effective prevention for your HAE attacks. But what exactly does that mean?
KIM: I can answer that! In a clinical study that lasted 6.5 months and included 125 people with HAE aged 12 years and older, TAKHZYRO was clinically proven to help reduce HAE attacks by 87% on average compared with placebo.
LINDA: I remember my doctor telling me that TAKHZYRO also helps reduce the frequency and severity of HAE attacks.
KIM: That's right, Linda! People in this clinical study also had an average of 83% fewer moderate or severe attacks and 87% fewer attacks requiring on-demand treatment compared with placebo.
LINDA: You know, I’ve experienced periods of time when I had no attacks while taking TAKHZYRO!
KIM: You’re not alone! In the same 6.5-month clinical study, 44% of people taking TAKHZYRO had zero attacks during the entire study compared with 2% taking placebo.
LINDA: Should we mention that TAKHZYRO was also looked at in a long-term, open-label extension clinical study?
KIM: Yes! That study lasted 2.5 years and included 212 people with HAE aged 12 years and older. It looked at the long-term safety and efficacy of TAKHZYRO. And patients knew they were receiving TAKHZYRO, which could have influenced the results. This second study showed similar and consistent results to the first study. If you’d like to learn more about the data, check out TAKHZYRO.com/studyresults. But, Linda, what does 'effective prevention' mean in your own words?
LINDA: For me, effective prevention comes down to 3 things: A significant reduction in the frequency and severity of my HAE attacks. Experiencing periods of freedom from HAE attacks. And freedom from daily dosing, with just 1 dose every 2 weeks.
KIM: Now that we know more about the idea behind effective prevention, let’s talk about dosing.
LINDA: As you know, TAKHZYRO is taken once every 2 weeks as a subcutaneous injection after you’ve been trained by a healthcare professional. Subcutaneous injection means you inject it under your skin.
KIM: In the clinical trial, most people taking TAKHZYRO were able to self-inject in 1 minute or less.
KIM: You’ll also have options of where you’d like to administer TAKHZYRO; there are 3 locations: your abdomen, thigh, or upper arm.
LINDA: Being able to switch injection sites is really important to me. That training session helped me feel confident about injecting myself and set me up for success moving forward.
KIM: This is just the beginning of your journey: the first step toward helping to prevent HAE attacks with TAKHZYRO.
KIM: Hey, Linda. I think now is a good time to talk about setting goals and expectations with your medical team. It's an important part of your HAE management plan, which now includes treatment with TAKHZYRO.
LINDA: That’s a great idea, Kim!
KIM: You and your doctor have decided to make TAKHZYRO a part of your HAE management plan to help you achieve your personal treatment goals.
LINDA: If you haven't already, think about what expectations you have for your HAE management plan.
KIM: What are your overall treatment goals? Where do you picture yourself on this journey a year from now? Take some time to write down answers to the following questions: How often do you experience HAE attacks? How severe are they? How often do you use on-demand medication?
KIM: Logging this information will allow you to keep track of your HAE attacks and plan future goals. Because the 2020 US HAEA guidelines recommend reassessing your goals with your doctor periodically, it’s important to share this information. That way you can both see how you’re doing with your HAE management plan.
LINDA: When I was starting on TAKHZYRO, I discussed my HAE experience with my doctor. We talked about what it was like living with the unpredictability of my HAE attacks. We looked at the frequency of my HAE attacks and how often I was treating them with on-demand medication. We also talked about what I hoped to achieve by taking TAKHZYRO. He encouraged me to keep track of my HAE attacks so that I could look back and see how far I’d come after being on TAKHZYRO for a few months. In the end, that initial discussion was so important to me because it helped establish a starting point for my treatment journey. And helped me understand what to expect along the way.
KIM: Thanks, Linda! Collaborating with your doctor is one of the most important steps at the start of your treatment journey with TAKHZYRO.
KIM: Hi! When deciding to start TAKHZYRO, you may have heard of OnePath Product Support, a program that offers patients a range of services. Let’s talk a little about that and what they do. But before I begin, I’d like to introduce someone who's going to help me out.
ANDREW: Thanks, Kim! I’m Andrew, a patient ambassador for TAKHZYRO. I'll be sharing some of the experiences I’ve had with the OnePath team and how they helped me get started with TAKHZYRO.
KIM: When you're prescribed TAKHZYRO, your doctor will help fill out a Start Form, which will enroll you in the program. Soon after enrollment, you'll receive a call from your Patient Support Manager, or PSM, from the main OnePath phone number. They will act as your primary point of contact during your treatment journey. Save this number to your phone so you can recognize calls from OnePath in the future!
KIM: Your PSM will work with you one-on-one to make sure you have access to your prescribed medication. They’ll also help with the following: Working with your insurance provider to help make sure your treatment is covered. Enrolling you in the OnePath Co-Pay Assistance Program, if you qualify. Arranging for a trained nursing professional to teach you how to self-inject TAKHZYRO at home, or virtually.
ANDREW: Admittedly, the early stages of my journey were a bit overwhelming. But my PSM was there to help alleviate some of that stress.
KIM: When it comes to insurance, your PSM and the rest of the OnePath team will walk you through the process step by step. They’ll also help you understand what’s covered and what’s not, and what to do next. Their goal is to help make it all a little easier.
ANDREW: I was initially denied TAKHZYRO, but OnePath Product Support worked with my insurance company and doctor to get me approved and started on treatment.
ANDREW: They also addressed any questions and concerns I had about accessing TAKHZYRO. In fact, my PSM continues to offer support throughout my journey. It’s good to know you’re not alone in this.
KIM: OnePath will also work with your specialty pharmacy to make sure your medicine is delivered. These pharmacies help coordinate many aspects of care for patients taking medications that have specialized handling, storage, and distribution requirements, just like TAKHZYRO.
ANDREW: When I was approved for TAKHZYRO, it took some time to receive my first delivery from the specialty pharmacy. But that’s normal. My OnePath team was able to get in touch with the pharmacy to help. And they let me know if there would be any changes to the delivery of my medication. Best of all, this support is provided to you free of cost.
KIM: Now let’s talk about at-home injection training. This is extremely important, as you should never try to inject TAKHZYRO without being properly trained by a healthcare professional. Your PSM will call you to explain how that process works. First, a nurse will come to your house Then, they will show you how to inject your TAKHZYRO and stay with you while you do it. Lastly, they’ll help answer any questions you may have about the administration process.
KIM: Andrew, what was your experience with injection training?
ANDREW: The nurse who came to my house told me about the 3 areas where I could inject, which I was happy to learn about. She showed me and my wife how to prepare the medication and how to inject it. The injection itself took about a minute. That training session really set me up for success! Beforehand, I wasn’t quite sure how confident I would be with self-injections.
ANDREW: With OnePath Product Support, you’re not alone when you start your journey on TAKHZYRO. In the end, it was great to have the OnePath team on my side, especially during those first few months of treatment, when I wasn’t quite sure what to expect.
KIM: OnePath has been helping people with HAE for over 10 years—from helping with the insurance process to making sure you have consistent access to medication and at-home injection training.
KIM: Hey, Linda! I think it’s a good time to talk about the most common side effects of TAKHZYRO.
LINDA: Sounds good, Kim! It’s really important for people to understand the side effects associated with any medication they take.
KIM: When we say “most common side effects,” we’re talking about the side effects that were experienced by most people in the clinical trial. These were injection site reactions, upper respiratory infection (an example of this is when someone gets a cold), headache, rash, muscle pain, dizziness, and diarrhea. You can see the percentage of patients that experienced these side effects in the table. Today let’s talk about injection site reactions.
KIM: If you’d like to read more about the other side effects of TAKHZYRO, you can visit the website URL shown here.
LINDA: Kim, some people may not know what injection site reactions are. Can you explain them?
KIM: Of course! Injection site reactions are most commonly pain, redness, or bruising in the area where TAKHZYRO was injected. In the clinical trial, 52% of the 84 people who received TAKHZYRO reported injection site reactions.
KIM: These reactions are expected, though.
LINDA: When I first started TAKHZYRO, I wasn’t sure how I felt about injecting myself. Would it hurt? Would I have a reaction? Thankfully, my doctor reassured me. I was informed that it may hurt a bit, but to keep taking TAKHZYRO exactly as directed.
LINDA: My PSM set up an at-home injection training session for me. It made me feel confident injecting my medication moving forward.
KIM: Glad you brought that up! If you haven't already, you'll be trained how to inject TAKHZYRO by a healthcare professional before your first dose. Be sure to use that time to ask any questions you may have about the injection process.
KIM: And remember! Call your doctor right away, or get emergency help, if you experience any of the following symptoms of an allergic reaction while taking TAKHZYRO: wheezing, difficulty breathing, chest tightness, fast heartbeat, faintness, rash, or hives.
KIM: Just as everyone’s experience with HAE is different, every person will respond to medication differently. That's why it's important to understand what the side effects of TAKHZYRO are, and what you can do if you experience them.
KIM: Hey, Linda and Andrew! Today I want to talk about staying on track with TAKHZYRO and steady state. Do you remember what steady state is?
ANDREW: Hi, Kim! It’s been a while, but I remember my doctor telling me that it’s when the amount of TAKHZYRO in the body becomes constant.
KIM: That’s right. Starting with your first dose of TAKHZYRO, the medicine builds in your body over time. Over the course of your first 6 doses, the amount of TAKHZYRO in your body will reach a constant level that is known as steady state. That’s why it’s important to give TAKHZYRO about two and a half months to reach steady state.
KIM: Keep in mind that TAKHZYRO is a medication that continues to work as you take it. That means you need to take it as prescribed by your doctor and avoid missing or skipping doses.
KIM: Once you start taking TAKHZYRO, it’s important to know that breakthrough HAE attacks can still occur.
ANDREW: I started experiencing fewer HAE attacks shortly after beginning treatment. But not all patients are the same. Experiences, and expectations, may vary. Right, Linda?
LINDA: Of course. When I was starting out on TAKHZYRO, I was still experiencing HAE attacks. To be honest, I was disappointed. I had expected fewer attacks after starting treatment. So I called my doctor. He reassured me right away that this was normal. He explained that the medicine needed time to build up in my body. Once I understood that, I was convinced I needed to give it time and keep taking TAKHZYRO. After a few months, I noticed my HAE attacks were less frequent and less severe than they had been before. I was proud of myself for sticking to my plan.
KIM: So if a breakthrough attack happens, don’t be discouraged. Talk to your doctor about your goals and expectations with preventive treatment, as well as the importance of continuing to take TAKHZYRO as prescribed.
KIM: Like Linda, you may notice that over time your HAE attacks are less frequent and less severe or that you are using your on-demand treatment less. These are signs that TAKHZYRO is working!
LINDA: If you have any questions about steady state or the clinical study data as you begin to take TAKHZYRO, speak with your doctor, like I did!
KIM: Remember, in order for TAKHZYRO to work effectively, you need to keep taking it as prescribed.
KIM: Andrew, Linda, let’s talk about patients staying on TAKHZYRO for the long term. After all, we want their HAE management plan to be successful!
LINDA: Sounds great, Kim!
KIM: We’ll discuss follow-up visits with the doctor and tips on how to remember to take TAKHZYRO.
KIM: As you get comfortable with your TAKHZYRO routine, it’s important to revisit the treatment goals you and your doctor made at the start of your journey.
KIM: Ask yourself this: Have the frequency and severity of your HAE attacks changed in the months since you’ve started TAKHZYRO? If they have, talk to your doctor about those changes and what your next set of goals may be.
LINDA: Setting goals helped me see how I was doing with my HAE management plan.
LINDA: It helps to have realistic goals for your treatment. For me, I wanted fewer and less severe HAE attacks. So when I started taking TAKHZYRO, I tracked 3 things: The frequency of my attacks. Their severity. And how often I used my on-demand treatment. Once I started to see progress, my doctor and I decided TAKHZYRO was the right choice for me.
ANDREW: At this point in your journey, it’s important to have these moments of reflection. These are the moments that helped me realize the bigger picture: my life before and after I started preventive treatment with TAKHZYRO. Moments like these showed me how much TAKHZYRO has helped and can continue to help manage my HAE. Since starting TAKHZYRO, my HAE attacks have been less frequent and less severe. I've gone several months without an attack! I’ve come a long way since the beginning of my journey. All thanks to choosing effective prevention with my doctor.
KIM: Remember, TAKHZYRO continues to work as you take it, so it’s important to keep taking it as prescribed by your doctor. You should also avoid skipping and missing doses.
KIM: Takeda is committed to helping you stay on track with your treatment.
LINDA: It’s true! The TAKHZYRO Text Reminder Program has been a big help when it comes to taking my medication on schedule. Just text SIGNUP to 36395 to start receiving reminders.
KIM: You can get more information about the Text Reminder Program by visiting the TAKHZYRO website.
KIM: In addition to the Text Reminder Program, what are some other ways you remember to take your TAKHZYRO as prescribed?
ANDREW: On my dosing days, right before my wife and I start our morning errands, she reminds me to take my TAKHZYRO injection. Injecting takes about a minute. And it’s something that I’ve gotten used to. Hopefully, you will too!
LINDA: I like to take my TAKHZYRO right after my morning cup of coffee on injection days. It’s easy for me to remember it that way.
KIM: Andrew, Linda, can you share what long-term prevention has meant for you?
LINDA: I like knowing I’m taking a medication that has been studied for as long as it has. Plus, TAKHZYRO has been prescribed to over 2,500 people. Being one of those people is reassuring. It's also reassuring to see the results I've had with TAKHZYRO.
ANDREW: I like the fact that I can inject TAKHZYRO and then not think about it again until my next dose. TAKHZYRO has helped me have fewer attacks, allowing me to reimagine my possibilities.
KIM: Your journey may be just beginning, but know you're part of a growing community that recognizes the importance of having a preventive treatment as part of their HAE management plan. You have the tools to get started and stay on track with TAKHZYRO. Now it's your turn to draw the next chapter of your journey. Keep exploring TAKHZYRO.com for more information as well as stories from patients like you.
RECORDING: TAKHZYRO® (lanadelumab-flyo) is a prescription medicine used to prevent attacks of hereditary angioedema (HAE) in people 2 years of age and older. It is not known if TAKHZYRO is safe and effective in children under 2 years of age. Important safety information. TAKHZYRO may cause serious side effects, including allergic reactions. Call your healthcare provider or get emergency help right away if you have any of the following symptoms: wheezing, difficulty breathing, chest tightness, fast heartbeat, faintness, rash, hives. The most common side effects seen with TAKHZYRO were injection site reactions, pain, redness, and bruising; upper respiratory infection, headache, rash, dizziness, diarrhea, and muscle aches. These are not all the possible side effects of TAKHZYRO. For more information, ask your healthcare provider or pharmacist. You may report side effects to FDA at 1-800-FDA-1088. TAKHZYRO has not been studied in pregnant or breastfeeding women. Talk to your healthcare provider about the risk of taking TAKHZYRO if you are pregnant, plan to be pregnant, are breastfeeding, or plan to breastfeed.
TAKHZYRO On-Demand Webinars
Learn more about HAE and TAKHZYRO from these informative webinars.
Choosing the Path of Prevention: TAKHZYRO & Children
View Transcript
More Info
This webinar video is designed to inform parents and caregivers about TAKHZYRO as a preventive treatment option for children.
Find out how TAKHZYRO could help.
Takeda Host Renee Seigel: Hi everyone. Thanks so much for joining. As mentioned, I am Renee Siegel. I am a patient marketer with Takeda, and I am happy to be back here as your host for today's webinar.
Takeda has been dedicated to advancing care in the hereditary angioedema space for more than a decade, and we are proud to be able to support the community in a holistic way. Our webinars with the HAEA are a part of that commitment.
In case anyone here is new to the subject, I would like to quickly set the stage. HAE is a rare genetic disease that causes attacks of swelling that can be painful, unpredictable, debilitating, and it affects about one in 50,000 people worldwide.
It involves reoccurring attacks of severe swelling of various parts of the body, including the hands, feet, genitals, abdomen, face, and throat. Swelling in the airway can restrict breathing and that can be fatal.
People with HAE do not have enough C1-INH protein or the C1-INH they do have does not work as well as it should. This causes another protein called kallikrein to become overactive, which results in the excessive release of an attack producing substance known as bradykinin.
The results of this excessive release are fluid leakage and swelling, otherwise known as an HAE attack. An HAE attack can start in one location and then it can spread to another before resolving.
Now getting into the topic of today's webinar, which is Choosing the Path of Prevention, TAKHZYRO and Children - as many of you are aware, there are two types of treatment for HAE.
On demand treatments are used to treat the symptoms of an HAE attack, and preventive treatments are medicines that you can take routinely to help prevent or reduce the frequency and severity of HAE attacks.
This is a really exciting and an important step forward for HAE treatment for younger patients.
TAKHZYRO is now a treatment for a broad range of children and adults with HAE, and it has been prescribed to more than 3250 people since 2018. Since launching TAKHZYRO for pediatrics, we are so excited to see pediatric patients and families beginning to choose TAKHZYRO as a treatment option. We are just so excited to be able to fulfill this need and to show up for the community in this way.
You will hear tonight from both a doctor and a person living with HAE, as previously introduced, who will provide information in their perspectives on having a prevention mindset when it comes to treating HAE.
First, I would like to review the important safety information about TAKHZYRO before we get into this conversation.
What is TAKHZRYO? TAKHZYRO or lanadelumab-flyo, is a prescription medicine used to prevent attacks of hereditary angioedema in people two years of age and older. It is not known if TAKHZYRO is safe and effective in children under two years of age.
TAKHZYRO may cause serious side effects, including allergic reactions. Call your healthcare provider or get emergency help right away if you have any of the following symptoms: wheezing, difficulty breathing, chest tightness, fast heartbeat, faintness, rash, or hives.
The most common side effects seen with TAKHZYRO were injection site reactions such as pain, redness and bruising, upper respiratory infection, headache, rash, dizziness, diarrhea, and muscle aches.
These are not all the possible side effects of TAKHZYRO. For more information, ask your healthcare provider or pharmacist. You may report side effects to the FDA at 1-800-FDA-1088.
TAKHZYRO has not been studied in pregnant or breastfeeding women. Talk to your healthcare provider about the risk of taking TAKHZYRO if you are pregnant, plan to be pregnant, are breastfeeding, or plan to breastfeed. Please see the full prescribing information, including information for patients available at this presentation.
How is HAE impacting your child?
HAE is an unpredictable, debilitating and potentially life-threatening disease. The onset of HAE symptoms can occur at any age, but studies suggest that symptoms may start to appear during childhood. Children with HAE can be impacted in many areas of their life, and the family can be impacted as well.
As mentioned earlier, the HAEA guidelines recommend always having access to on demand treatment and that long-term prevention may be considered for anyone with HAE.
With all of that information, I am now really happy to introduce Dr. Joshua Jacobs, who is an allergy and immunology specialist treating HAE in adults, adolescents and children. He is currently practicing in California.
HCP Speaker Dr. Joshua Jacobs: Thank you, Renee. Glad to be here.
Takeda Host Renee Seigel: So happy to have you. I am also really excited to welcome one of our wonderful long time patient speakers and advocates in the HAE community, Bobbi. Bobbi lives with HAE herself and has two daughters who were diagnosed as children with HAE and are now adults.
Bobbi has years of experience managing her own HAE, being involved in the community, and caring for her daughters when they were children with HAE, and she very kindly agreed to participate in today's webinar.
Patient Ambassador Bobbi P.: Thank you so much, Renee, and hello to all of our attendees. Renee, how about we start with a poll?
Takeda Host Renee Seigel: Yeah, I think that is a great idea, Bobbi. You will have a poll that will pop up on the screen. This is our first question. Who here cares for or helps care for a child with HAE?
If you are a friend of the family, that counts as well. We know everyone here knows that all the support counts and it takes a village. We will give you a second to answer this poll.
From the poll, it actually looks like the majority of the participants here do not help or care for a child with HAE. So, it is really interesting to know that you're here to learn more about this information which is exciting to see.
Patient Ambassador Bobbi P.: My two daughters have HAE. My oldest was diagnosed when she was in fifth grade and my youngest in kindergarten. We have all been through a lot together.
It is great to see that there's a lot of people on here that want to learn more about taking care of children with HAE.
Every adult and child with HAE are different and HAE is so unpredictable so it really can affect any and every area of your life.
Takeda Host Renee Seigel: Yes, that is interesting. Bobbi. Can you tell us a little bit more?
Patient Ambassador Bobbi P.: Sure. Both of my daughters had different experiences with HAE. My oldest was affected a lot more than my youngest with frequent attacks, especially at school.
She went from never missing a day of school to struggling to just have a good day.
It was non-stop educating the school and her teachers about HAE, working with the home health tutor, and just trying to maintain her learning and her schedule.
Ultimately, we had to pull her out of school so she could concentrate on her health.
This was a very difficult and sad decision for us, but we knew that in the long term this was the best decision. She really had her life up ended and struggled with anxiety and depression as well.
HCP Speaker Dr. Joshua Jacobs: That sounds really hard, Bobbi. I have heard similar stories from other families I've worked with.
The predictability of HAE can be difficult for patients and families to deal with. I have children with HAE suffer from attacks in various parts of the body; face, hands, feet, even laryngeal or throat attacks which can be life threatening. These attacks can be different for each individual and can change over time.
Takeda Host Renee Seigel: Yes. Thank you both for that input and let me just jump in for a second to ask another question for our attendees.
I know the majority of you answered that you don't currently care for a child with HAE, but for those that do - Where has HAE most affected the child that you do care for?
We are going to take a moment so that you can answer this question. While everyone answers, I would like to share some examples from a parent that we recently spoke with. Her name is Ada. Ada and her daughter Kenzleigh both have HAE, and they have generously agreed to share some of their stories and experiences with us, which has been helpful and interesting.
When Kenzleigh was about five or six years of age, Ada noticed swells in Kenzleigh's hands and then a swell that started in her eye and eventually moved to her throat.
Kenzleigh had to be intubated, which some of you might imagine was extremely scary for both Ada and Kenzleigh.
When reflecting on the story with Ada, Ada said that she now looks back on this and feels like it is a reminder that attacks are unpredictable, and you can never really know what the next attack will look like.
After Kenzleigh was formally diagnosed with HAE, they both would try to predict when attacks would come. For example, when she bumped herself, fell, or was upset or stressed, but you just never know and could never really predict it.
Patient Ambassador Bobbi P.: Those experiences are powerful and definitely remind me of what it was like when my girls were growing up. I remember back before my daughters and I were on TAKHZYRO, it was really hard for my family, and especially when my oldest was having throat swells on a regular basis, or when all three of us were having an attack at the same time.
Takeda Host Renee Seigel: Yes, definitely.
Well thank you, Bobbi, for sharing that. Why don't we take a second so we can pull up the results in the poll?
So, it looks like for “where has HAE most affected your child?” It actually looks like it's across the board. It is split between at school, at home, socializing, and then actually the majority of you, 70%, said all of the above, so all these areas.
Just kind of reflecting back on what Bobbi was sharing and just in general, the reflections - I agree that these stories are so powerful, but only approved routine preventive treatment options for children 6 to less than 12 years of age required dosing every three to four days. Children with HAE two to less than six years of age, had no approved preventative treatment, making TAKHZYRO the first preventive treatment for this age group.
Dr. Jacobs, would you mind telling everyone a bit about the studies that led to this?
HCP Speaker Dr. Jacobs: I would love to, Renee. It is great news for the HAE community. So really, we are going to start here and we're going to talk about the initial trials for TAKHZYRO HELP trials. The first clinical trial for TAKHZYRO was indeed term to HELP, and this is the clinical trial that led to FDA approval for TAKHZYRO.
It was a 6 1/2-month trial. It included 125 people diagnosed with HAE type one and two, aged 12 years and older. The main goal of the study was to evaluate the ability of TAKHZYRO 300 mg every two weeks to reduce the frequency of HAE attacks. In this study, people taking TAKHZYRO had 87% fewer attacks than those on placebo.
Injection site reactions were the most common side effects of TAKHZYRO in the 6 1/2-month study, with people aged 12 years and older. The next study was completed after FDA approval of TAKHZYRO, and we call this the HELP OLE. OLE stands for open label extension.
Open label means that everyone in this study is getting medicine, and they know what they are receiving. From the standpoint of effect, there may be some influence on results.
This was again a 2 1/2-year study of 212 people diagnosed with HAE aged 12 years and older. The main goal of the study was to evaluate the long-term safety, and that was with TAKHZYRO 300 mg every two weeks. In this study, people taking TAKHZYRO for an average of 2 1/2 years had similar results to those in the 6 1/2-month clinical trial. The most common side effects seen in this long-term open label study were injection site reactions, and those included pain, redness and bruising, upper respiratory infections, and rash.
Now the TAKHZYRO pediatric study is called the Spring Study. It was the largest pediatric trial of any preventive treatment in HAE, and there were twenty-one children in the 52-week open label study, all aged between 2 and 11 years.
The main goals were one, to evaluate the safety of TAKHZYRO 150 mg taken every two weeks or every four weeks, and two, to measure levels of TAKHZYRO in the body for children 2 to 11 years of age. So, in the study, children from two years to six years of age got one dose every four weeks, and children from 6 to 11 got one dose every two weeks. Overall, the safety and tolerability of TAKHZYRO were similar between adults and pediatric patients. 2 to 17 years of age, or 12 to 17 years of age. There were no serious side effects reported. No one discontinued due to the side effects and there were no allergic reactions related to TAKHZYRO.
The most common treatment emergent adverse events in children taking TAKHZYRO 150 mg every two or four weeks were injection site pain, redness and swelling, administration site pain and injection site reactions, again were the most common.
We do need to point out that TAKHZYRO can cause serious side effects in some people, including allergic reactions, and if that happens, people should call their child's healthcare provider or get emergency help. Use of TAKHZYRO in children 2 to 11 years of age was supported by the efficacy data in the 6 1/2-month study.
For people 12 years of age and older, additional data showed that similar levels of TAKHZYRO were reached in the body for adults and children.
A secondary goal of the study was to measure the ability of TAKHZYRO 150 mg taken once every two weeks or four weeks to prevent HAE attacks in children 2 to 11 years of age. Decrease in HAE attack was measured as the number of attacks before the patient started the study, compared to the number of HAE attacks after taking TAKHZYRO.
The study was not designed to understand how well TAKHZYRO works in children. The twenty-one children included knew they were taking TAKHZYRO, and this did not compare TAKHZYRO to another product or a placebo. These details make it difficult to determine how well TAKHZYRO decreased HAE attacks in children. On average, children had 95% fewer HAE attacks compared to before starting the study, and 76% of children were attack free for the entire 52 weeks, or one year of the study.
Patient Ambassador Bobbi P.: Thanks so much for sharing that information, Dr. Jacobs, I am just so excited for our HAE community.
Takeda Host Renee Seigel: Yes, me too.
Patient Ambassador Bobbi P.: I cannot wait to see how it might help the community of children and their caregivers. I was in the clinical trials for TAKHZYRO, as were my daughters, and we started going weeks and then months without an attack. There were tears of joy and it was just overwhelming.
HCP Speaker Dr. Jacobs: That is so great to hear, Bobbi. I noticed the same kind of relief in my pediatric patients who were included in the most recent clinical trial.
Takeda Host Renee Siegel: Yes, and Kenzleigh was also in the pediatric clinical trial, the Spring Study, as Dr. Jacobs mentioned. Ada told us that she wanted to start Kenzleigh on TAKHZYRO as soon as she could because she was having so many face and throat swells. Ada is very proactive and wanted to do as much as she could to help prevent Kenzleigh from having attacks, which for her, TAKHZYRO was a part of that mindset.
Ada says, we have had discussions that TAKHZYRO has really helped manage Kenzleigh's HAE attacks. Before she enrolled in the study, she was having attacks every week.
Patient Ambassador Bobbi P.: So, Dr. Jacobs, I know a lot of people are going to be thinking about injections and how they can make them more comfortable for kids. Can you give us some tips around that?
HCP Speaker Dr. Jacobs: Absolutely can, Bobbi.
Yes, the frequency of the dose depends on the age of the child, right? So, every four weeks for kids two to less than six, and every two weeks for kids six and older. When treating kids, we do think a lot more about how they will handle the treatment itself and we also would expect kids not to love injections. I am 100% on board with creating a reward system for children who are taking TAKHZYRO. This can work for a child, or really a person of any age. For example, after your injection, we will go to the playground, get some ice cream, whatever works best for your particular child. My mantra is comfort and confidence. Do whatever helps to make the child comfortable physically and emotionally and feel confident in the medication.
Help the child create a positive mindset. Talk to the child about the injection, the reason for it, and how TAKHZYRO is going to help them.
Obviously, the way to do that will depend on the child's age and prior knowledge. They may have seen you or another family member administer an injection for example. I would also suggest that you rotate the injection sites. There are three places mentioned in the instructions for use that comes with TAKHZYRO.
Patient Ambassador Bobbi P.: Oh yes, I am very familiar with those, stomach, thigh, and upper arm.
Which for my daughters and I, we usually inject in our stomachs. That is our preference.
HCP Speaker Dr. Jacobs: Exactly. And so, then I would suggest choosing the environment thoughtfully, somewhere the child feels comfortable and safe, and of course, somewhere clean. It can help to make it the same time and place every time if the child seems calm and willing. We want to normalize the experience, make it ordinary and not frightening, and always be encouraging.
Takeda Host Renee Siegel: Definitely. Thank you both for sharing those thoughts and perspectives and from Ada's perspective, she said that she was apprehensive at first, but that they have a good routine going. It takes just a few minutes every two weeks and Kenzleigh is grateful to be taking a medication that helps reduce the frequency of HAE attacks.
Patient Ambassador Bobbi P.: TAKHZYRO was not available when my girls were young, but I think it's such an exciting step for the HAE community.
Takeda Host Renee Siegel: Yes. Based on what we call or reference specialty pharmacy data, TAKHZYRO is currently the number one prescribed preventative treatment in HAE.
We are looking forward to helping even more, and as I mentioned, so excited to see we're beginning to help this population with this approval for this expanded age range.
HCP Speaker Dr. Jacobs: TI would like to also add here that ongoing family conversations are important. Kids grow up, family dynamics change, priorities around activities change, and it is important to be consistent and get the proper dose at the right intervals.
Takeda Host Renee Siegel: Yes, definitely. There is a resource where people can sign up. They can sign up for dosing reminders which could help, especially if something in the family routine as you are referencing Dr. Jacobs changes.
HCP Speaker Dr. Jacobs: Yes, it is crucial to remember that TAKHZYRO is not a cure, and to take it as prescribed at regular intervals. I hate to see patients having breakthrough attacks, but it can happen and is something they need to be prepared for.
Patient Ambassador Bobbi P.: I want to second your suggestion about having conversations not just with your family, but with your healthcare team and anyone else who you might consider part of your circle about everything that is going on. A dear friend of ours who lives close by, was so supportive and valuable to our family during those tough years. I cannot thank her enough for all the time she was there to help with whatever was needed.
I was also so grateful for all the people I have met through our HAE community - they were and still are my rock and sounding board.
HCP Speaker Dr. Jacobs: Indeed, I have open and honest relationships with all my patients.
Takeda Host Renee Siegel: That is great. Speaking of support, Takeda has more than 12 years of experience supporting the HAE community. I want to remind everyone here that the OnePath program is available. You can access a dedicated patient service manager support manager who offers injection training, financial assistance options, and product support. Let us also not forget that caregivers need support too, whether they have HAE or not. Caregiving can be very demanding, as some of you might know, and we have been talking about children with HAE today, and for every child with HAE, there is a caregiver who could use support as well, and OnePath offers support for caregivers too.
Patient Ambassador Bobbi P.: Yes, that is a great thing.
I would also like to tell caregivers that it is OK to give yourself time and not feel guilty about it with younger kids especially. You are their board, your voice, and do not give up. As a parent, you just want them to flourish.
You need to take care of yourself so that you can take care of others. I know it is not always an easy thing to do.
You can also lean on this community. I have spent the last 10+ years participating in numerous activities, and I will continue to do everything I can to support our community. I encourage everyone to get involved.
Takeda Host Renee Sigel: Thank you, Bobbi. You are an inspiration.
We are also really excited to take a few minutes to hear from all of you. But before that, I just wanted to say that there have been more than 3250 people prescribed TAKHZYRO and in addition their caregivers, friends and families are part of our community too.
So, before I hand it back to the HAEA to take some questions, I just want to take a moment to thank Bobbi and Dr. Jacobs, and although they could not be here tonight, I also want to thank Ada and Kenzleigh for allowing us to share their experiences and story.
Choosing the Path of Prevention: TAKHZYRO & Children
View Transcript
Takeda Host Renee Seigel: Hi everyone. Thanks so much for joining. As mentioned, I am Renee Siegel. I am a patient marketer with Takeda, and I am happy to be back here as your host for today's webinar.
Takeda has been dedicated to advancing care in the hereditary angioedema space for more than a decade, and we are proud to be able to support the community in a holistic way. Our webinars with the HAEA are a part of that commitment.
In case anyone here is new to the subject, I would like to quickly set the stage. HAE is a rare genetic disease that causes attacks of swelling that can be painful, unpredictable, debilitating, and it affects about one in 50,000 people worldwide.
It involves reoccurring attacks of severe swelling of various parts of the body, including the hands, feet, genitals, abdomen, face, and throat. Swelling in the airway can restrict breathing and that can be fatal.
People with HAE do not have enough C1-INH protein or the C1-INH they do have does not work as well as it should. This causes another protein called kallikrein to become overactive, which results in the excessive release of an attack producing substance known as bradykinin.
The results of this excessive release are fluid leakage and swelling, otherwise known as an HAE attack. An HAE attack can start in one location and then it can spread to another before resolving.
Now getting into the topic of today's webinar, which is Choosing the Path of Prevention, TAKHZYRO and Children - as many of you are aware, there are two types of treatment for HAE.
On demand treatments are used to treat the symptoms of an HAE attack, and preventive treatments are medicines that you can take routinely to help prevent or reduce the frequency and severity of HAE attacks.
This is a really exciting and an important step forward for HAE treatment for younger patients.
TAKHZYRO is now a treatment for a broad range of children and adults with HAE, and it has been prescribed to more than 3250 people since 2018. Since launching TAKHZYRO for pediatrics, we are so excited to see pediatric patients and families beginning to choose TAKHZYRO as a treatment option. We are just so excited to be able to fulfill this need and to show up for the community in this way.
You will hear tonight from both a doctor and a person living with HAE, as previously introduced, who will provide information in their perspectives on having a prevention mindset when it comes to treating HAE.
First, I would like to review the important safety information about TAKHZYRO before we get into this conversation.
What is TAKHZRYO? TAKHZYRO or lanadelumab-flyo, is a prescription medicine used to prevent attacks of hereditary angioedema in people two years of age and older. It is not known if TAKHZYRO is safe and effective in children under two years of age.
TAKHZYRO may cause serious side effects, including allergic reactions. Call your healthcare provider or get emergency help right away if you have any of the following symptoms: wheezing, difficulty breathing, chest tightness, fast heartbeat, faintness, rash, or hives.
The most common side effects seen with TAKHZYRO were injection site reactions such as pain, redness and bruising, upper respiratory infection, headache, rash, dizziness, diarrhea, and muscle aches.
These are not all the possible side effects of TAKHZYRO. For more information, ask your healthcare provider or pharmacist. You may report side effects to the FDA at 1-800-FDA-1088.
TAKHZYRO has not been studied in pregnant or breastfeeding women. Talk to your healthcare provider about the risk of taking TAKHZYRO if you are pregnant, plan to be pregnant, are breastfeeding, or plan to breastfeed. Please see the full prescribing information, including information for patients available at this presentation.
How is HAE impacting your child?
HAE is an unpredictable, debilitating and potentially life-threatening disease. The onset of HAE symptoms can occur at any age, but studies suggest that symptoms may start to appear during childhood. Children with HAE can be impacted in many areas of their life, and the family can be impacted as well.
As mentioned earlier, the HAEA guidelines recommend always having access to on demand treatment and that long-term prevention may be considered for anyone with HAE.
With all of that information, I am now really happy to introduce Dr. Joshua Jacobs, who is an allergy and immunology specialist treating HAE in adults, adolescents and children. He is currently practicing in California.
HCP Speaker Dr. Joshua Jacobs: Thank you, Renee. Glad to be here.
Takeda Host Renee Seigel: So happy to have you. I am also really excited to welcome one of our wonderful long time patient speakers and advocates in the HAE community, Bobbi. Bobbi lives with HAE herself and has two daughters who were diagnosed as children with HAE and are now adults.
Bobbi has years of experience managing her own HAE, being involved in the community, and caring for her daughters when they were children with HAE, and she very kindly agreed to participate in today's webinar.
Patient Ambassador Bobbi P.: Thank you so much, Renee, and hello to all of our attendees. Renee, how about we start with a poll?
Takeda Host Renee Seigel: Yeah, I think that is a great idea, Bobbi. You will have a poll that will pop up on the screen. This is our first question. Who here cares for or helps care for a child with HAE?
If you are a friend of the family, that counts as well. We know everyone here knows that all the support counts and it takes a village. We will give you a second to answer this poll.
From the poll, it actually looks like the majority of the participants here do not help or care for a child with HAE. So, it is really interesting to know that you're here to learn more about this information which is exciting to see.
Patient Ambassador Bobbi P.: My two daughters have HAE. My oldest was diagnosed when she was in fifth grade and my youngest in kindergarten. We have all been through a lot together.
It is great to see that there's a lot of people on here that want to learn more about taking care of children with HAE.
Every adult and child with HAE are different and HAE is so unpredictable so it really can affect any and every area of your life.
Takeda Host Renee Seigel: Yes, that is interesting. Bobbi. Can you tell us a little bit more?
Patient Ambassador Bobbi P.: Sure. Both of my daughters had different experiences with HAE. My oldest was affected a lot more than my youngest with frequent attacks, especially at school.
She went from never missing a day of school to struggling to just have a good day.
It was non-stop educating the school and her teachers about HAE, working with the home health tutor, and just trying to maintain her learning and her schedule.
Ultimately, we had to pull her out of school so she could concentrate on her health.
This was a very difficult and sad decision for us, but we knew that in the long term this was the best decision. She really had her life up ended and struggled with anxiety and depression as well.
HCP Speaker Dr. Joshua Jacobs: That sounds really hard, Bobbi. I have heard similar stories from other families I've worked with.
The predictability of HAE can be difficult for patients and families to deal with. I have children with HAE suffer from attacks in various parts of the body; face, hands, feet, even laryngeal or throat attacks which can be life threatening. These attacks can be different for each individual and can change over time.
Takeda Host Renee Seigel: Yes. Thank you both for that input and let me just jump in for a second to ask another question for our attendees.
I know the majority of you answered that you don't currently care for a child with HAE, but for those that do - Where has HAE most affected the child that you do care for?
We are going to take a moment so that you can answer this question. While everyone answers, I would like to share some examples from a parent that we recently spoke with. Her name is Ada. Ada and her daughter Kenzleigh both have HAE, and they have generously agreed to share some of their stories and experiences with us, which has been helpful and interesting.
When Kenzleigh was about five or six years of age, Ada noticed swells in Kenzleigh's hands and then a swell that started in her eye and eventually moved to her throat.
Kenzleigh had to be intubated, which some of you might imagine was extremely scary for both Ada and Kenzleigh.
When reflecting on the story with Ada, Ada said that she now looks back on this and feels like it is a reminder that attacks are unpredictable, and you can never really know what the next attack will look like.
After Kenzleigh was formally diagnosed with HAE, they both would try to predict when attacks would come. For example, when she bumped herself, fell, or was upset or stressed, but you just never know and could never really predict it.
Patient Ambassador Bobbi P.: Those experiences are powerful and definitely remind me of what it was like when my girls were growing up. I remember back before my daughters and I were on TAKHZYRO, it was really hard for my family, and especially when my oldest was having throat swells on a regular basis, or when all three of us were having an attack at the same time.
Takeda Host Renee Seigel: Yes, definitely.
Well thank you, Bobbi, for sharing that. Why don't we take a second so we can pull up the results in the poll?
So, it looks like for “where has HAE most affected your child?” It actually looks like it's across the board. It is split between at school, at home, socializing, and then actually the majority of you, 70%, said all of the above, so all these areas.
Just kind of reflecting back on what Bobbi was sharing and just in general, the reflections - I agree that these stories are so powerful, but only approved routine preventive treatment options for children 6 to less than 12 years of age required dosing every three to four days. Children with HAE two to less than six years of age, had no approved preventative treatment, making TAKHZYRO the first preventive treatment for this age group.
Dr. Jacobs, would you mind telling everyone a bit about the studies that led to this?
HCP Speaker Dr. Jacobs: I would love to, Renee. It is great news for the HAE community. So really, we are going to start here and we're going to talk about the initial trials for TAKHZYRO HELP trials. The first clinical trial for TAKHZYRO was indeed term to HELP, and this is the clinical trial that led to FDA approval for TAKHZYRO.
It was a 6 1/2-month trial. It included 125 people diagnosed with HAE type one and two, aged 12 years and older. The main goal of the study was to evaluate the ability of TAKHZYRO 300 mg every two weeks to reduce the frequency of HAE attacks. In this study, people taking TAKHZYRO had 87% fewer attacks than those on placebo.
Injection site reactions were the most common side effects of TAKHZYRO in the 6 1/2-month study, with people aged 12 years and older. The next study was completed after FDA approval of TAKHZYRO, and we call this the HELP OLE. OLE stands for open label extension.
Open label means that everyone in this study is getting medicine, and they know what they are receiving. From the standpoint of effect, there may be some influence on results.
This was again a 2 1/2-year study of 212 people diagnosed with HAE aged 12 years and older. The main goal of the study was to evaluate the long-term safety, and that was with TAKHZYRO 300 mg every two weeks. In this study, people taking TAKHZYRO for an average of 2 1/2 years had similar results to those in the 6 1/2-month clinical trial. The most common side effects seen in this long-term open label study were injection site reactions, and those included pain, redness and bruising, upper respiratory infections, and rash.
Now the TAKHZYRO pediatric study is called the Spring Study. It was the largest pediatric trial of any preventive treatment in HAE, and there were twenty-one children in the 52-week open label study, all aged between 2 and 11 years.
The main goals were one, to evaluate the safety of TAKHZYRO 150 mg taken every two weeks or every four weeks, and two, to measure levels of TAKHZYRO in the body for children 2 to 11 years of age. So, in the study, children from two years to six years of age got one dose every four weeks, and children from 6 to 11 got one dose every two weeks. Overall, the safety and tolerability of TAKHZYRO were similar between adults and pediatric patients. 2 to 17 years of age, or 12 to 17 years of age. There were no serious side effects reported. No one discontinued due to the side effects and there were no allergic reactions related to TAKHZYRO.
The most common treatment emergent adverse events in children taking TAKHZYRO 150 mg every two or four weeks were injection site pain, redness and swelling, administration site pain and injection site reactions, again were the most common.
We do need to point out that TAKHZYRO can cause serious side effects in some people, including allergic reactions, and if that happens, people should call their child's healthcare provider or get emergency help. Use of TAKHZYRO in children 2 to 11 years of age was supported by the efficacy data in the 6 1/2-month study.
For people 12 years of age and older, additional data showed that similar levels of TAKHZYRO were reached in the body for adults and children.
A secondary goal of the study was to measure the ability of TAKHZYRO 150 mg taken once every two weeks or four weeks to prevent HAE attacks in children 2 to 11 years of age. Decrease in HAE attack was measured as the number of attacks before the patient started the study, compared to the number of HAE attacks after taking TAKHZYRO.
The study was not designed to understand how well TAKHZYRO works in children. The twenty-one children included knew they were taking TAKHZYRO, and this did not compare TAKHZYRO to another product or a placebo. These details make it difficult to determine how well TAKHZYRO decreased HAE attacks in children. On average, children had 95% fewer HAE attacks compared to before starting the study, and 76% of children were attack free for the entire 52 weeks, or one year of the study.
Patient Ambassador Bobbi P.: Thanks so much for sharing that information, Dr. Jacobs, I am just so excited for our HAE community.
Takeda Host Renee Seigel: Yes, me too.
Patient Ambassador Bobbi P.: I cannot wait to see how it might help the community of children and their caregivers. I was in the clinical trials for TAKHZYRO, as were my daughters, and we started going weeks and then months without an attack. There were tears of joy and it was just overwhelming.
HCP Speaker Dr. Jacobs: That is so great to hear, Bobbi. I noticed the same kind of relief in my pediatric patients who were included in the most recent clinical trial.
Takeda Host Renee Siegel: Yes, and Kenzleigh was also in the pediatric clinical trial, the Spring Study, as Dr. Jacobs mentioned. Ada told us that she wanted to start Kenzleigh on TAKHZYRO as soon as she could because she was having so many face and throat swells. Ada is very proactive and wanted to do as much as she could to help prevent Kenzleigh from having attacks, which for her, TAKHZYRO was a part of that mindset.
Ada says, we have had discussions that TAKHZYRO has really helped manage Kenzleigh's HAE attacks. Before she enrolled in the study, she was having attacks every week.
Patient Ambassador Bobbi P.: So, Dr. Jacobs, I know a lot of people are going to be thinking about injections and how they can make them more comfortable for kids. Can you give us some tips around that?
HCP Speaker Dr. Jacobs: Absolutely can, Bobbi.
Yes, the frequency of the dose depends on the age of the child, right? So, every four weeks for kids two to less than six, and every two weeks for kids six and older. When treating kids, we do think a lot more about how they will handle the treatment itself and we also would expect kids not to love injections. I am 100% on board with creating a reward system for children who are taking TAKHZYRO. This can work for a child, or really a person of any age. For example, after your injection, we will go to the playground, get some ice cream, whatever works best for your particular child. My mantra is comfort and confidence. Do whatever helps to make the child comfortable physically and emotionally and feel confident in the medication.
Help the child create a positive mindset. Talk to the child about the injection, the reason for it, and how TAKHZYRO is going to help them.
Obviously, the way to do that will depend on the child's age and prior knowledge. They may have seen you or another family member administer an injection for example. I would also suggest that you rotate the injection sites. There are three places mentioned in the instructions for use that comes with TAKHZYRO.
Patient Ambassador Bobbi P.: Oh yes, I am very familiar with those, stomach, thigh, and upper arm.
Which for my daughters and I, we usually inject in our stomachs. That is our preference.
HCP Speaker Dr. Jacobs: Exactly. And so, then I would suggest choosing the environment thoughtfully, somewhere the child feels comfortable and safe, and of course, somewhere clean. It can help to make it the same time and place every time if the child seems calm and willing. We want to normalize the experience, make it ordinary and not frightening, and always be encouraging.
Takeda Host Renee Siegel: Definitely. Thank you both for sharing those thoughts and perspectives and from Ada's perspective, she said that she was apprehensive at first, but that they have a good routine going. It takes just a few minutes every two weeks and Kenzleigh is grateful to be taking a medication that helps reduce the frequency of HAE attacks.
Patient Ambassador Bobbi P.: TAKHZYRO was not available when my girls were young, but I think it's such an exciting step for the HAE community.
Takeda Host Renee Siegel: Yes. Based on what we call or reference specialty pharmacy data, TAKHZYRO is currently the number one prescribed preventative treatment in HAE.
We are looking forward to helping even more, and as I mentioned, so excited to see we're beginning to help this population with this approval for this expanded age range.
HCP Speaker Dr. Jacobs: TI would like to also add here that ongoing family conversations are important. Kids grow up, family dynamics change, priorities around activities change, and it is important to be consistent and get the proper dose at the right intervals.
Takeda Host Renee Siegel: Yes, definitely. There is a resource where people can sign up. They can sign up for dosing reminders which could help, especially if something in the family routine as you are referencing Dr. Jacobs changes.
HCP Speaker Dr. Jacobs: Yes, it is crucial to remember that TAKHZYRO is not a cure, and to take it as prescribed at regular intervals. I hate to see patients having breakthrough attacks, but it can happen and is something they need to be prepared for.
Patient Ambassador Bobbi P.: I want to second your suggestion about having conversations not just with your family, but with your healthcare team and anyone else who you might consider part of your circle about everything that is going on. A dear friend of ours who lives close by, was so supportive and valuable to our family during those tough years. I cannot thank her enough for all the time she was there to help with whatever was needed.
I was also so grateful for all the people I have met through our HAE community - they were and still are my rock and sounding board.
HCP Speaker Dr. Jacobs: Indeed, I have open and honest relationships with all my patients.
Takeda Host Renee Siegel: That is great. Speaking of support, Takeda has more than 12 years of experience supporting the HAE community. I want to remind everyone here that the OnePath program is available. You can access a dedicated patient service manager support manager who offers injection training, financial assistance options, and product support. Let us also not forget that caregivers need support too, whether they have HAE or not. Caregiving can be very demanding, as some of you might know, and we have been talking about children with HAE today, and for every child with HAE, there is a caregiver who could use support as well, and OnePath offers support for caregivers too.
Patient Ambassador Bobbi P.: Yes, that is a great thing.
I would also like to tell caregivers that it is OK to give yourself time and not feel guilty about it with younger kids especially. You are their board, your voice, and do not give up. As a parent, you just want them to flourish.
You need to take care of yourself so that you can take care of others. I know it is not always an easy thing to do.
You can also lean on this community. I have spent the last 10+ years participating in numerous activities, and I will continue to do everything I can to support our community. I encourage everyone to get involved.
Takeda Host Renee Sigel: Thank you, Bobbi. You are an inspiration.
We are also really excited to take a few minutes to hear from all of you. But before that, I just wanted to say that there have been more than 3250 people prescribed TAKHZYRO and in addition their caregivers, friends and families are part of our community too.
So, before I hand it back to the HAEA to take some questions, I just want to take a moment to thank Bobbi and Dr. Jacobs, and although they could not be here tonight, I also want to thank Ada and Kenzleigh for allowing us to share their experiences and story.
This webinar video is designed to inform parents and caregivers about TAKHZYRO as a preventive treatment option for children.
Find out how TAKHZYRO could help.
This is my TAKHZYRO Experience: Imagine Yours
View Transcript
Takeda Host Kristi Christensen: Thank you, Troyce. First, I wanted to say hello! Good evening! Thank you for joining us tonight and thank you to the HAEA (Hereditary Angioedema Association) for hosting tonight's webinar and I appreciate all that information Troyce and I have the pleasure of introducing you to our two speakers tonight. If you do not already know these two, I am thrilled that I get to be the one to introduce you.
Dr. Daniel Soteres is out of Colorado Springs, Colorado. He is in private practice there and he will be our physician and immunologist speaker this evening.
Pam is joining us as our patient ambassador speaker, and she has been a speaker with us for six years. As we get started, just as a housekeeping reminder, even though Troyce did mention it, we are going to hold any questions that come in over Q&A until the close of the program. If we do not address your specific question, the HAEA follows up after the program in the next day or two to answer those questions for you. At this point I am thrilled to turn it over to Dr. Soteres. Thank you so much for doing this tonight.
HCP Speaker Dr. Soteres:Hey, thanks y'all and it is a pleasure to be a part of this. It is great to have all of you all online with us.
TAKHZYRO is what we are discussing tonight. You are going to join 3250+ people who have chosen to prevent HAE (HEREDITARY ANGIOEDEMA) attacks before they happen.
That is all since 2018. So, what is TAKHZYRO? It is a prescription medicine used to prevent attacks of HAE in people two years of age and older. You heard that right. Two years of age and older. More on that later.
It's not known if TAKHZYRO is safe in those under two years of age.
There are, of course, safety slides throughout this presentation. Here is your introduction to that: TAKHZYRO may cause serious side effects like allergic reactions. Some of those symptoms might be wheezing, breathing difficulty, chest tightness, increased heart rate, faintness, rash, or hives.
I am Dr. Soteres. I'm a board-certified allergy immunologist in Colorado Springs. We have a Clinical Research Center here. I am a clinical professor on faculty with the University of Colorado Health Sciences Center. I have been a primary investigator for studies in a range of topics, specifically in HAE.
I've been doing clinical research trials. I date this by my son's age, he's 18. I have been doing this for about 17 years.
Here is my guiding principle: I try to consider the whole patient while treating all their health concerns. I should include that I am a speaker for Takada tonight. I have been a consultant with them, and I do get payment for this presentation, just to be clear.
These slides are not for reproduction and are limited to the scope of our presentation tonight. So, what we're going to cover today is HAE, what is it, the impact of HAE attacks, and how to work with your doctor to create a personalized HAE management plan.
You are also going to hear about TAKHZYRO from a real patient's experience. That's where Pam comes in. You're going to learn about a treatment option to help prevent HAE attacks in those two years and older.
Great. Next, we will go to Pam.
Patient Ambassador Pam: Hello everyone, I want to thank you for joining us for this webinar. My name is Pam and I have been a patient speaker for Takada since 2017 and I am so thankful that you have spent your valuable time tonight to join us for this webinar.
My story will be broken up into two parts, the first being about my experience living with HAE, and the second part will be about my life today, and my experiences managing my HAE with Takeda’s preventive medication, TAKHZYRO. You'll also see pictures of my life. The highs, the lows, and the people who matter the most to me. I promise to do my very best to explain who these people are, and what these photos mean. So, with that, let us get started.
This is going to be a little different; I'm not going to start at the beginning when my HAE presented itself. Rather, I want you to know and feel what my life has been like living with this condition. Now, of course I'm showing you this wedding picture because that was long before I knew that I had HAE, probably another decade before I actually was diagnosed. It was a long struggle to get to that point as I know it was for many of you.
I'm going to focus on two parts of my life, my career as a journalist, and my life raising my kids as a single parent. I am going to start with my career.
It was the late 80s and into the 90s where I enjoyed a career as a reporter before taking increasingly higher roles within the news department. I worked both in front of the camera as an on-air reporter and anchor, and behind as a news director.
Everywhere I looked and everything I saw and induced stress; stress from deadlines, stress from stories, wherever I went, it was just with me.
But you grow accustomed to it. It's like the drum beat of your life and you adapt. But no matter how comfortable I was within that environment, I also found myself battling HAE attacks due to the stress. I did not know what they were at the time, but too many times I found myself sitting in my car after a long day dealing with an attack, or the aftereffects of attacks.
The worst part about that period in time, was that my attacks were often abdominal. It was like a screwdriver being driven into my abdomen.
Staying upright and not wincing in pain was all I could do to get through the day, whether I was in the newsroom or out on assignment. Whenever these attacks happen, my focus would change. Just get through it and get to the weekend. Then I could rest.
I did everything in my power to overcome the challenges HAE presented me before and even after I was diagnosed in the early 1990s. I would be reporting on location from whatever story I'd be covering and hoping an abdominal attack would ease its grip on me between takes.
I'd use all my vacation time to cover the days I missed dealing with my attacks so nobody would question my dedication to the job. It was all I could do to protect my career. It was a career I loved from the invasiveness of this disease.
But in doing so, I also found myself struggling through obligations at home with my children.
I was a single mother, a widow to three young girls. My husband, who you see in these pictures with our daughters, in fact, the one in front of the fireplace, was taken, not very long before he passed away. So, the girls were still young.
After he died, I was alone grasping the life as a single parent with a demanding career and children who needed me.
HAE is persistent and completely unpredictable. It does not choose the right time or the right place. Usually, it's always the wrong time in the wrong place, and as much as I felt that at work, I felt it even more at home.
I didn't miss many important events, but there were some. One was an elementary open house. My daughter had been working on her project for weeks. I wasn't going to make it because of an abdominal attack.
She walked in the room, stood beside my bed, and burst into tears, saying, why can't you just go?
The guilt and helplessness I felt were a lot worse than the attack itself.
But I just couldn't bring myself to get out of bed. All I wanted to do was be there for her, but my attack, like the ones I've described already, just wouldn't let go.
It was like wave after wave of pain seized my stomach and I felt like I couldn't move, let alone be there for her. There was this feeling of shame, like I was letting everyone down around me. Shockingly, this wasn't the turning point in my journey with HAE.
That came when my oldest daughter was diagnosed with HAE. I really started learning about the disease, what to do for it, what the future held, and how to make it better for the next generation. All the aspects of living with HAE that I had ignored for so many years; the types of H AE attacks people experience that triggers management plan options, all of it became something I researched, discussed, and followed.
My first step was to reach out to the HAE community, which is how I found the US hereditary angioedema association, or as we know the, HAEA.
I have to tell you that my first HAEA meeting was amazing. I didn't have to explain what I had. We started talking about coping, what worked for others and how to improve life. I also realized that it was time to seriously consider treatment options. I wanted to set a good example for my children, and I needed to connect with a doctor who could help me manage my HAE. More importantly, I needed to have something that would allow me to live a life less impacted and controlled by my HAE attacks. I show you this picture from my wedding because after I was diagnosed, and started putting the pieces together, I did figure out where this had come from. My dad on the far side of the picture is who I had gotten HAE from, and then my grandmother is who he had gotten it from. Then of course, I passed it on to my two children. That is about as far as I could go back in the family to figure out where that family line had come with HAE.
So going back to the story, I decided to get my first HAE treatment and at that time it was an on-demand medication, which I had used for many, many years. This treatment was a lifeline for the life I was living. I finally had something to combat the attacks, particularly the abdominal attacks that were stopping me from being fully present in my kids' lives, my life, and work.
This was my first experience with any semblance of control with my HAE attacks and it felt great.
I had finally taken a step toward living with my HAE, rather than always enduring painful attack after painful attack.
But as the years went on, I saw myself battling the aftereffects of an attack despite using my acute treatment. Because using your on-demand medication means you are still having an HAE attack.
This after effect, the same effect I mentioned earlier that stole weekends and nights and moments from my family, pushed me toward prevention. What if I could treat the swells before they ever came? Thanks for listening to this first part of my story. I'll hand it back to Dr. Soteres before coming back to tell you a little more about my decision to take TAKHZYRO and how I'm doing today.
HCP Speaker Dr. Soteres: That’s a great story, Pam. Thank you for sharing that. I have so many thoughts. I can't wait till the question session to ask you my questions about some of the story.
We're going to move forward talking about the disease state. This is about hereditary angioedema tonight. It is a rare genetic condition affecting about one in 50,000 people.
What does that mean? I always try to put these numbers in perspective. Well, in the US, we have a population of about 300 million and for one in 50,000, we are talking about 6000 or 7000 people in the United States who have hereditary angioedema. Another way to put that in perspective is anybody ever found a four-leaf clover? Well, that is about one in 10,000.
HAE attacks are the result of an imbalance in blood proteins starting with C1 inhibitor, and this affects other chemicals like kallikrein and bradykinin.
Bob here, you see him pictured as a real patient with HAE and we see the impact from a facial attack of hereditary angioedema. Of course, HAE attacks can start in one location and spread to another. They can include areas like the face, the throat, abdomen, genitalia, hands, and feet.
HAE is unpredictable. I think Pam really outlined that quite well. It can change over time and your next attack may be nothing like your last attack. So unpredictable, debilitating, and potentially life threatening, those throat attacks can be quite a concern.
Past attacks do not predict future attacks, or whether the airway may become affected. Disease severity can change over time, so it's important to make a management plan with your doctor and review it regularly.
You're going to review your HAE management plan at each visit. I'm a doctor, I work in clinical practice, and I see close to 1500 patients a year. I love it when my patients come with a list of questions. I can tell you from personal experience. I'm a doctor and so is my wife. When my kids were young, and it was my turn to take him to the doctor's office, my wife would tell me all the questions I was supposed to ask. I would get there and forget them, and maybe remember some of them. Then I'd get home and she would say, 'what about this, this, and this?' I am like 'oh, I forgot to ask that one!'
Anyway, I think it is great to use this as an avenue to write down some of these and take these answers to your providers. If you want to make a difference on the impact that HAE has on your life, here are some topics to discuss with your doctor. On the left-hand side, you see topics to discuss at the first visit, the average number of attacks you experience per month, including the severity and location, whether you have missed work, school, or other activities due to an HAE attack and how often, the awareness of treatments and whether you've researched any, and how on demand and preventative therapies work. At your follow-up visits every visit thereafter; Again, average number of attacks per month including severity and location, how often you feel the need to use your on-demand treatment, how the unpredictability of HAE is affecting you, and how preventative treatment may impact the frequency and or severity of those attacks.
The 2020 US HAEA guidelines recommend a personalized approach to HAE management. Patients should focus on two general areas of treatment. One is on-demand treatment, the other is long-term preventative treatment. In the on-demand category, we're thinking patients should have access to at least two doses of their on-demand therapy, and every attack should be considered for treatment. Long-term preventative therapy should be considered for every patient based on their individual needs. You're going to review your HAE management plan regularly with your doctor and discuss if you should be considered for long-term preventative treatment.
Stop and think; what are some of the ways that HAE has impacted your life in the past 6 to 12 months? Here are some thoughts; for starters, the unpredictability of attacks - I will give you an example. I love to mountain bike; it is a thing I do for fun. One of my HAE patients likes to mountain bike also. I was talking with her one afternoon, just following up on the phone with how she was doing. She said, 'oh, I had to miss a mountain bike trip with my friends because I had an HAE attack' and I said, 'hold on a second, I thought you said you weren't having a lot of attacks?' She's like, 'well, you know, I didn't really count that one.' We ended up in a whole conversation about how maybe the disease is affecting more of your day-to-day and week-to-week decisions, work life, recreation than you originally thought. So many people are used to just managing it saying, 'I'll just deal with it this time.' But the bigger picture might be a little more than the person next to you is having to manage.
How often have you had attacks, and what is the severity of your attacks, and have you had any hospital visits? That of course is an important data point.
The 2020 US HAEA guidelines recommend TAKHZYRO as one of the first line therapies for long-term prevention. I should say that at your presentation and certainly available online and many sources, you can get the prescribing information for TAKHZYRO. This includes the full list of indications and safety as well.
So, we have a poll question. What is most important to you when considering a preventative treatment?
We have chosen one. We're going to talk about these. Oh, wow., 88% are in favor of long-term attack reduction. 13% were concerned about the safety profile. The other options: dosing schedule, how the treatment is administered, and patient support services, those were zero. The main one long-term attack prevention and then in second place safety profile. Thank you.
Indications and important safety information. TAKHZYRO is a prescription medicine used to prevent attacks of hereditary angioedema in people two years of age and older. It is not known if it's safe and effective in patients younger than two years of age.
It may cause serious side effects; we have already reviewed these at the very beginning. This is the list of allergic reaction concerns, wheezing, breathing difficulties, chest tightness, increased heart rate, faintness, rash, and hives.
Remember, the most common side effects seen with TAKHZYRO have really been injection site reactions, which would be pain, redness and bruising at the site of the location, upper respiratory infection, headache, rash, dizziness, diarrhea, and muscle aches. We see a list of contact numbers here and if there are concerns about side effects, you would want to be talking with your prescribing. healthcare provider, and you can also report those to the FDA at this 1-800 number.
Additionally, TAKHZYRO has not been studied in pregnant or breastfeeding women. Talk to your healthcare provider about the risk of taking TAKHZYRO if you're pregnant or planning to become pregnant, or you are breastfeeding or planning to breastfeed.
There is a broad range of people with HAE. TAKHZYRO is the number one prescribed HAE preventative treatment. People who've experienced TAKHZYRO include children, adolescents, and adults with HAE, people with mild moderate or severe hereditary angioedema, and more than 3250 people have been prescribed TAKHZYRO since 2018.
The warnings on here we have already reviewed, so that is great. We can move forward.
TAKHZYRO offers adolescents and adults proven HAE attack reduction, the possibility of 0 attacks for up to a year, and one subcutaneous self-injection every two weeks.
In a 6 1/2-month clinical trial of 125 people with HAE 12 and older, TAKHZYRO reduced the number of monthly attacks compared to placebo.
People taking TAKHZYRO also had fewer moderate or severe attacks, and less attacks requiring acute treatment compared with the placebo group as well.
They extended the study for 2 1/2 years. That is an open label, so no more placebo control. This was 212 people 12 and older with HAE. Some of those people had zero attacks for up to a year as well.
Imagine a life with fewer HAE attacks. The first study is what led to the FDA approval of TAKHZYRO. That was the 6 1/2-month clinical research trial with 125 people 12 and older. The main goal of the study was the ability of TAKHZYRO to reduce the frequency of HAE attacks. On average, people taking TAKHZYRO 300 mg every two weeks had much fewer attacks and less severity of their attacks. 44% of the people had zero attacks.
More details from this 6 1/2-month study, people had an average of 83% fewer moderate or severe attacks compared to the placebo group, and 87% had fewer attacks requiring on-demand treatment compared with placebo.
The second study was completed after FDA approval. This is the 2 1/2-year open label extension study with 212 people, each of them 12 years or older with HAE.
The main goal of this study was to evaluate the long-term safety of TAKHZYRO, 300 mg every two weeks. These people knew they were receiving TAKHZYRO, and of course that can influence the study results. On average, 87% of people had fewer attacks compared with their baseline number of attacks, and 69% of people had zero attacks for up to a year.
So, freedom from daily dosing with TAKHZYRO. It takes less than one minute to self-inject and it is dosed every two weeks.
There are three choices for injection site. The middle column is 2 weeks between each dose, and on the right are 3 choices for injection site. So, the slide, if you can imagine, reads 123, less than one minute to self-inject, 2 weeks between each dose and three choices for injection site.
We have reviewed the warnings at the bottom here so we can move forward to the next slide.
This is an important point, reaching steady state for people 12 years of age and older. What do we mean by steady state? With the first dose of TAKHZYRO, the medicine will start to build up in the body and over the first six doses, TAKHZYRO will reach a constant level, also known as steady state.
We see those in in the graphic there on the bottom, the doses are numbered 1 2 3 4 5 6 and seven. We see that around 2 1/2 months. That's the two-week dosing dose 6. That's when we're at steady state in the body.
TAKHZYRO safety results have been established in one of the largest prevention studies in HAE.
While it may cause serious side effects, including allergic reactions, you're going to contact your healthcare providers or get emergency help for that situation. Injection sites were really the most common side effect of TAKHZYRO. If we look at the safety information and the graphic on the right, we look across the top and we see column one; the most common side effects in the study, and then in the middle column we see TAKHZYRO in red there, and the placebo column. We look at injection site reactions for the first row and we see 52% for TAKHZYRO, and about 34% for the placebo. We can follow that graphic down and we see the upper respiratory infections that are very similar to placebo at 29% and 32%, headache at 21 and 22%, rash 7 and 5%. Then we see dizziness at 6% and zero. We see diarrhea and muscle aches as well.
In the 2 1/2-year Open label extension trial, remember this one was specifically focused on the safety data, the most common side effects seen in the long-term open label study were injection site reactions, upper respiratory infection, and headache.
We see the side effects listed in the left-hand column, and TAKHZYRO 300 mg every two weeks in the right-hand column: Injection site pain, upper respiratory infections, headaches. Then we see some joint pain, back pain, diarrhea, sinus concerns, influenza, nausea, and urinary tract infections. When my patients have concerns about the side effects of medications, I really look at it as an opportunity to have a discussion. It doesn't mean that the drug isn't right for you or it's not working. It might just be a barrier to overcome. For me, I love it when they come in with a list of questions. If these are on your mind, you should be writing these down and discussing them with your healthcare provider who prescribes this to further your conversation and knowledge.
TAKHZYRO was studied in children. Safety was the main goal of the study. The study included 21 children with HAE two to less than 12 years of age and taking 1 subcutaneous injection every two or four weeks. The safety profile was evaluated in a 52-week open label. That means no placebo in the study of 21 children with HAE from 2 to less than 12 years old.
The recommended dosage is 150 mg every two weeks for children from 6 to 12 years old and the dosage is every four weeks for those two to six years old.
The ability of TAKHZYRO to prevent HAE attacks in children was measured as a secondary objective of the study.
So, safety was established in the largest pediatric trial of any preventative treatment in HAE. In the 52-week study, there were no serious side effects reported, no discontinuations due to the side effects, and no allergic reactions related to TAKHZYRO.
Overall, the safety and tolerability of TAKHZYRO were similar between the adult and the pediatric patients. The most common side effects seen in the 12-month open label Pediatric study were again, the injection site reactions with pain and redness listed. We have a very similar graphic on the right-hand side here. We see the most common related TEAEs treatment and emergent adverse event. Then we see the TAKHZYRO with the dosing 2- or 4-weeks injection site pain, redness, swelling at administration, site pain, and site reactions.
The effectiveness of TAKHZYRO in children was a secondary goal of the 52-week study, and that was to measure the ability of TAKHZYRO to prevent HAE attacks in children two to less than 12 years old.
The efficacy data from the 6 1/2-month study in people 12 and older and data from the 52-week study showed similar levels of TAKHZYRO were reached in the body for adults and children. This was another main goal of the 52-week trial.
There are a few things to note about this study when considering efficacy. The study was not designed to understand how well TAKHZYRO works in children. The 21 children included knew they were taking TAKHZYRO. Again, there was no placebo. For comparison, these details make it difficult to determine how well TAKHZYRO decreased HAE attacks in children.
On average, children had 95% fewer HAE attacks compared to before starting the study. 76% of children were attack free for the entire 52-week study.
Dosing that fits in your child's life when it comes to your child's HAE treatment; it helps if the dosing schedule works with their lifestyle. Self-administration is not recommended for those two to less than 12 years old and the recommended dosages again for children two to less than six are 150 mg every four weeks for children 6 to less than 12, 150 mg every two weeks. If your child is taking TAKHZYRO every two weeks and it's well controlled, meaning they have zero HAE attacks for more than six months, their doctor may consider switching them to every four-week dosing.
Ada shares her daughter's experience with TAKHZYRO. Isn't she cute? I like Kenzleigh here. I'm just going to read through this, and I'm not going to change my voice at all, so forgive me. There's a lot on the page, but we will just walk through this together and you all can enjoy Kenzleigh’s cute photos. What made you decide to start Kenzleigh on TAKHZYRO? 'Well, my doctor told me that Kenzleigh might be eligible to take part in a TAKHZYRO study for children.
I was familiar with TAKHZYRO because my sister takes it, and it works for her. I knew right away I wanted to start her on it, especially after seeing the clinical studies. Kenzleigh was having face and throat swells. It was clear that her on demand treatment was not enough. We are so grateful that the TAKHZYRO study came along at the right time for her.
How has Kenzleigh responded to TAKHZYRO? So far TAKHZYRO has helped manage Kenzleigh 's HAE attacks. We know we can rely on TAKHZYRO, and my hope is that she can continue to take it for as long as she needs.
What advice do you have for patients of children with HAE? Do not give up. Your child needs to be strong. Work with your doctor to find the solution that works best for your child. Be brave, be present and be knowledgeable about disease and medicine.'
Start to imagine your TAKHZYRO experience. HAE attacks can be unpredictable, debilitating and potentially life threatening.
In a 6 1/2-month clinical study for people 12 years of age and older, taking TAKHZYRO 300 mg every two weeks, people experienced 87% fewer attacks on average compared with placebo.
Freedom from daily dosing; once every two weeks for people six years of age and older, or every four weeks for people two to less than six years of age. Remember, more than 3250 people in the US have been prescribed TAKHZYRO since 2008.
All right, we are back to Pam.
Patient Ambassador Pam: Thanks Dr. Soteres
Dr. Soteres is talking about TAKHZYRO, but in my story, we were back where I was just starting to take the on-demand medication, and that would really have gotten me in the fight. I was finally able to experience some relief from my HAE attacks. But like I said, what comes after an attack was still there. So as time went on, the novelty of treating with an on-demand medication only kind of wore off and I started to see all the ways my HAE attacks were still impacting my life.
I kept wondering, could we do more?
It was not a single attack that made the idea of prevention interesting to me. It was years of attack after attack after attack that made me realize the importance of what if.
What if I did not experience some of those attacks? What if I had a chance to live a life with fewer days stuck in the wake of an attack?
That is right around the time my doctor introduced me to TAKHZYRO. My doctor and I were drawn to the efficacy data and the idea of me possibly experiencing fewer and less severe future attacks.
We discussed how TAKHZYRO works to control the flow of bradykinin in the body, which is the substance that causes our painful attacks, and I was instantly drawn to it. And as you can see, my family is growing at this point. I did want to be more available because now we have new little ones that we have got to run after and take care of. I want to be active with them too.
I like the idea of taking TAKHZYRO once every two weeks because I could see that as a good fit for my lifestyle. We also make sure to discuss the potential risks and I think Dr. Soteres has mentioned all these allergic reactions as well as some more common ones like injection site reactions, upper respiratory infections, and headaches. Those are all-important factors to consider when you are deciding how to treat your HAE.
After discussing all of those important points, my doctor and I decided it was time to move forward with TAKHZYRO and take a preventive approach to managing my HAE.
For all that went into my decision to start TAKHZYRO, my first preventative treatment, I am happy to be here and tell you it has been a good option for me.
I have consistently gone months between attacks, and I even went a year between early 2019 and early 2020 without a single attack. It is surreal to say that aloud. I never thought that that would be possible. These results have given both my doctor and I confidence in how we are managing and possibly preventing future attacks. Of course, these are my experiences and not everyone will be the same, so please make sure you work with your doctor to see if it would be a good option for you.
Accepting my condition and accepting that I needed to take treating my HAE seriously was a life changing shift for me.
It is something that would not have been possible if I had not taken the leap to become more involved with this community. It is like I had lived alone in a dark room, afraid of what was outside the walls that I had built up around myself, only to realize the support I needed was just on the other side.
Fear is such a powerful thing; it can stop us from becoming the people we dream of being. I do not want to look back on my life and see what has happened to me or my daughters. In the moment you are most afraid, remember, you are in charge of how you manage your HAE and how you engage with the HAE community.
You have the power to decide what is best for you. There will still be moments when you feel overwhelmed by this disease, but there's so much hope and progress in the HAE community.
I want the same for all of you - live life on your own terms. Educate yourself. Reach out for support from others, and never lose hope.
Thanks again for being here.
Takeda Host Kristi Christensen: We roll right into another poll question.
On behalf of Takeda and the marketing team, I get the opportunity to talk about additional resources that we provide, and this is perfect because Dr. Soteres led us right up for this. The first resource is a doctor discussion guide. If you would like to see your healthcare provider prepared, you can use that discussion guide to guide those conversations and ask questions like, how many attacks have you had in the last six months and ask you to rate the severity of those attacks just for an example. The next thing is the YouTube channel where you can hear from other patients with HAE about their experiences. One of those patients actually is Pam. You can see Pam on the YouTube channel talking about her experience.
Next is the toolkit. A lot of people like to have something to get them started. So, this includes an attack tracker, a medical ID card and more.
Then there is a text reminder program. If any of you are like me, I rely on text reminders daily for almost everything in my life. The text reminder program will remind you when those two weeks are coming up and it is time to take your next dose of TAKHZYRO.
Here we go. This is the poll question.
It looks very similar to the first poll question.
We will see if anything has changed since Dr. Soteres presented all our clinical trial information and we talked about resources.
We started with an 88% long term attack reduction. This has shifted just a little bit, and safety has become maybe even a little bit more important.
Thank you for that.
The next thing is our OnePath product support. We are very fortunate to have a few ways that OnePath can help along your journey. The first one is a dedicated patient support manager. Your PSM (Patient Support Manager) is a single point of contact that you will have through your entire journey to help you with any TAKHZYRO support needs that you may have.
Next is financial assistance options. When it comes to finding out about things like copay assistance for those who are eligible, or any assistance that you need navigating any kind of insurance coverage, OnePath is there to help you with those things. Injection training: when you get started on TAKHZYRO, you may need someone to teach you how to do that.
OnePath will set up in home training from a nurse that is trained just to teach you how to give your injection or to help you teach a caregiver how to do that for you. Then the last thing is resources and education. Connecting you and your family members with educational resources, including the OnePath mobile app.
You can see that there is a QR code here if you would like to scan it, you can learn more about the things that are offered through one path through that link.
Then, what to expect from OnePath? That toolkit that we discussed on the resources slide will be shipped to you from OnePath and your PSM and your one path team will facilitate a benefits investigation. They will make a call to your insurance company to find out what your coverage will look like for TAKHZYRO and then figure out from there what kind of financial and coverage options you have.
Then TAKHZYRO will be delivered by a specialty pharmacy directly to you and not to your doctor, but to your home.
Watch an HAE expert and a person living with HAE talk about the impact of HAE's unpredictable attacks, why TAKHZYRO could help prevent them, and how you can work with your doctor to include TAKHZYRO in your management plan.
Find out how TAKHZYRO can help.