TAKHZYRO CLINICAL STUDY RESULTS

Freedom from HAE attacks: Some people experienced up to one year attack free

TAKHZYRO offers the possibility of fewer attacks based on results from clinical studies. In the studies, some patients experienced zero attacks over periods of time.

Dennis

First study leading
to
FDA approval
of TAKHZYRO

The 6.5-month clinical study included 125 people diagnosed with HAE aged 12 years and older. The main goal of the study was to evaluate the ability of TAKHZYRO 300 mg every 2 weeks to reduce the frequency of HAE attacks.

On average, people had

87%

fewer attacks

compared with placebo

of people

HAD ZERO
ATTACKS

for the entire 6.5-month
study
compared with 2%
of those
taking placebo

People also had 83% fewer moderate or severe attacks and 87% fewer attacks that needed acute treatment compared to placebo.

Second study
completed after
FDA
approval

The 2.5-year open-label extension clinical study included 212 people diagnosed with HAE aged 12 years and older. The main goal of this study was to evaluate the long-term safety of TAKHZYRO 300 mg every 2 weeks. Patients knew they were receiving TAKHZYRO, which could have influenced the study results.

In this study, people taking TAKHZYRO for an average of 2.5 years had similar results to those in the 6.5-month clinical study.

On average, people had

87%

fewer attacks

compared with baseline

of people

HAD ZERO
ATTACKS

for up to a year

People also had 84% fewer moderate or severe attacks and 93% fewer attacks that needed acute treatment compared with baseline.

Baseline means a person's attack rate before beginning treatment in either the clinical study or the long-term, open-label study.

All data presented are for TAKHZYRO 300 mg every 2 weeks unless otherwise indicated.

Safety results established in one of the
largest prevention studies in HAE

TAKHZYRO may cause serious side effects, including allergic reactions. Call your healthcare provider or get emergency help right away if you have symptoms of an allergic reaction.

Side effects displayed in the following tables occurred in ≥10% of people taking TAKHZYRO.

Tell your healthcare provider if you have any side effect that bothers you or that does not go away. These are not all the possible side effects of TAKHZYRO.

For more information, ask your healthcare provider or pharmacist.

The 6.5-month clinical study

Injection site reactions were the most common side effects of TAKHZYRO in the clinical study.

Most common side effects

TAKHZYRO
(84 people)*

Placebo
(41 people)

Injection site reactions

52%

34%

Pain

43%

29%

Redness

10%

Bruising

Upper respiratory infection

29%

32%

Headache

21%

22%

Rash

Muscle pain

Dizziness

Diarrhea

*Included all people treated with TAKHZYRO (300 mg every 2 weeks, 300 mg every 4 weeks, or 150 mg every 4 weeks) in the clinical study.

The 2.5-year, open-label extension study

The most common side effects seen in the long-term, open-label study included injection site reactions (pain, redness, and bruising), upper respiratory infections, and headache.

Most common side effects

TAKHZYRO 300 mg every 2 weeks (212 people)

Injection site pain

47%

Viral upper respiratory tract infection

42%

Upper respiratory tract infection

26%

Headache

25%

Injection site redness

17%

Joint pain

13%

Injection site bruising

12%

Back pain

12%

Diarrhea

11%

Sinus infection

11%

Influenza

10%

Nausea

10%

Urinary tract infection

10%

TAKHZYRO was also studied in children

The largest pediatric trial of any preventive treatment in HAE

The 52-week, open-label study included 21 children diagnosed with HAE aged 2 to <12 years. The main goals of the study were to:

Evaluate the safety profile of TAKHZYRO 150 mg taken once every 2 weeks or every 4 weeks
Measure the levels of TAKHZYRO in the body for children 2 to <12 years of age

Patients and their caregivers knew they were receiving TAKHZYRO, which could have influenced the results.

In the 52-week study, there were:

No serious side effects reported
No discontinuations due to side effects
No allergic reactions related to TAKHZYRO

In addition, levels of TAKHZYRO in the body for children 2 to <12 years of age who received 150 mg every 2 or 4 weeks were similar to those in adult patients receiving TAKHZYRO 300 mg every 2 weeks.

Most common related TEAEs in children taking TAKHZYRO 150 mg every 2 or 4 weeks (21 children)

Injection site pain: 29%
Injection site redness: 14%
Injection site swelling: 5%

Administration site pain: 5%
Injection site reaction: 5%

TEAE=treatment-emergent adverse event

TAKHZYRO may cause serious side effects, including allergic reactions. Call your healthcare provider or get emergency help right away if your child has symptoms of an allergic reaction.

The effectiveness of TAKHZYRO in children

Use of TAKHZYRO in children 2 to <12 years of age was supported by:

Efficacy data from the 6.5-month study in people 12 years of age and older
Additional data that showed similar levels of TAKHZYRO were reached in the body for adults and children

A secondary goal of the 52-week study was to measure the ability of TAKHZYRO 150 mg taken once every 2 weeks or once every 4 weeks to prevent HAE attacks in children 2 to <12 years of age. Decrease in HAE attacks was measured as the number of attacks before the patient started in the study compared to the number of HAE attacks after taking TAKHZYRO.

An important note about this study

The study was not designed to understand how well TAKHZYRO works in children. The 21 children included knew they were taking TAKHZYRO. This study did not compare TAKHZYRO to another product or 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
76% of children were attack free for the entire 52-week study

I was experiencing 2 to 3 attacks per month. Now with TAKHZYRO, I’ve gone up to 12 months without an HAE attack.

Bob Real TAKHZYRO patient

View Transcript

KELLY: I stay on treatment because I remember what my HAE was like before prevention, and I do not want to go back there.

JACK: HAE attacks are unpredictable. TAKHZYRO has made my HAE attacks less frequent. And let's face it, having fewer HAE attacks is just as important today as it was when I started treatment. I definitely don't want to go back to a life with more HAE attacks.

DENNIS: Injecting TAKHZYRO once every two weeks is certainly better than dealing with all the HAE attacks I used to have.

ANDREW: There were times before I started TAKHZYRO when all I could do was take my on-demand medication and push through the HAE attack, waiting for the medicine to work. Now that I'm having fewer attacks, I don't have to do that as much.

SORAYA: I can't say enough about the OnePath Product Support staff. They have been with me through the whole process. Whenever there was a problem accessing my TAKHZYRO, they will say, we are here to help you, and they always did. They work very hard to make sure I got my TAKHZYRO.

SCOTT: Sometimes, I would have HAE attacks three times a week. I was often missing out on plans, thanks to all that unpredictability. But with TAKHZYRO as part of my HAE management plan, my attacks have decreased, and I don't have to miss out on plans as often.

NARRATOR: TAKHZYRO (lanadelumab) is a prescription medicine used to prevent attacks of hereditary angioedema, HAE, in people 12 years of age and older. It is not known if TAKHZYRO is safe and effective in children under 12 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, and/or hives.

NARRATOR: The most common side effects seen with TAKHZYRO were injection site reactions, pain, redness, and bruising; upper respiratory infection; and headache. 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.

NARRATOR: Talk to your healthcare provider about TAKHZYRO, the only preventive HAE treatment you take as a subcutaneous injection just once every two weeks.

Explore more information on TAKHZYRO safety profile and side effects.

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