TAKHZYRO Efficacy
Studied in one of the largest prevention studies in HAE with the longest active treatment duration1-5
Studied in one of the largest prevention studies in HAE with the longest active treatment duration1-5
TAKHZYRO was studied in a range of patients with HAE type I or II1,2
HELP Study
- 70% Female6
- 30% Male6
- 12 to 73 Years of Age7
of active treatment
- (26 weeks)1,6*
with long-term
preventive therapy1
- 48% were on C1-INH alone6
- 3.2% were on oral therapy6†
- 4.8% were on both6
*One month was defined as 28 days in the clinical trial. †Androgens or antifibrinolytics.
Help Study Design
The clinical trial was a multicenter, double-blind, parallel group, placebo-controlled, dose-ranging study, which assessed the safety and efficacy of TAKHZYRO in 125 patients with HAE type I or II (≥12 years of age). Patients were randomized to receive TAKHZYRO 150 mg every 4 weeks (n=28), TAKHZYRO 300 mg every 4 weeks (n=29), TAKHZYRO 300 mg every 2 weeks (n=27), or placebo (n=41) for 26 weeks (6.5 months, where 1 month was defined as 28 days). Prior to randomization, patients ≥18 years of age were required to complete a ≥2-week long-term prophylaxis washout period. All patients then entered a 4-week run-in period to determine the baseline HAE attack rate. Patients with ≥1 investigator-confirmed HAE attack during the run-in period were eligible for study enrollment and randomization. The primary efficacy endpoint was the rate of investigator-confirmed attacks during the treatment period (time frame: from Day 0 to Day 182).1,6
Rediscover prevention
Significant reduction in mean attack rate‡ vs placebo at 6.5 months (26 weeks) in the pivotal trial1,6
- TAKHZYRO every 4 weeks resulted in a 73% reduction in attacks vs placebo (Adjusted P<0.001)1§
- Mean monthly attack rate at baseline (during run-in period): 3.52 for TAKHZYRO every 2 weeks (n=27); 3.71 for TAKHZYRO every 4 weeks (n=29); 4.02 for placebo (n=41)6
- Mean monthly attack rate (during treatment): 0.26 for TAKHZYRO every 2 weeks; 0.53 for TAKHZYRO every 4 weeks; 1.97 for placebo1
‡Mean monthly attack rate: number of attacks/4 weeks.
§Adjusted P-values for multiple testing.
with TAKHZYRO 300 mg every 2 weeks2
Attack reduction was consistently greater for patients taking TAKHZYRO vs placebo regardless of previous prophylaxis use, history of laryngeal attacks, or attack rate during run-in period.1
SELECT IMPORTANT SAFETY INFORMATION
Hypersensitivity reactions have been observed. In case of a severe hypersensitivity reaction, discontinue TAKHZYRO administration and institute appropriate treatment.
All secondary endpoints of the pivotal trial were met1,6
||Adjusted P values for multiple testing.
Many patients had zero attacks1,6
In a post hoc, exploratory analysis, after 6 doses (10 weeks) of TAKHZYRO (300 mg every 2 weeks):
PATIENTS
HAD ZERO ATTACKS
FOR THE NEXT 4 MONTHS
OF TREATMENT6¶
77% of patients taking TAKHZYRO every 2 weeks (n=26) had zero attacks vs 3% of patients taking placebo (n=37) (Day 70 to Day 182).6
- 45% of patients taking TAKHZYRO every 4 weeks (n=29) had zero attacks vs 3% with placebo (n=37) during steady-state period (Day 70 to Day 182)6¶
Based on a prespecified exploratory analysis, over the entire 6.5-month study duration, 44% of patients taking TAKHZYRO every 2 weeks (n=27) had zero attacks vs 2% of patients taking placebo (n=41) (Day 0 to Day 182).1,6¶
- 31% of patients taking TAKHZYRO every 4 weeks (n=29) had zero attacks vs 2% with placebo (n=41 for placebo) during the entire 6.5-month study (Day 0 to Day 182)1,6¶
¶Percentage of patients who had zero attacks over the entire 26-week study duration was a prespecified, exploratory endpoint. Percentage of patients who had zero attacks during the steady-state period (Day 70 to Day 182) was a post hoc analysis.
About 97% of patients in the HELP Study enrolled in the 2.5-year extension study2
HELP Open-label Extension Study
- 109 rollover patients, which represents about 97% of patients in the pivotal trial2
- 103 nonrollover patients2
- 81.6% of patients completed the study or enrolled in commercial product2
of active treatment2
- Patients were given TAKHZYRO 300 mg every 2 weeks for a mean duration of 29.6 (SD=8.2) months2
with long-term
preventive therapy2
- 50% were on C1-INH alone
- 6.1% were on oral therapy*
- 3.3% were on both
Long-term safety of TAKHZYRO was the primary endpoint in the open-label extension study.2
HELP Open-label Extension Study
The open-label extension study evaluated the safety and efficacy of TAKHZYRO for up to 2.5 years in patients with HAE type 1 or type II (≥12 years of age). It included patients from the pivotal trial (rollover n=109) and additional patients (nonrollover n=103). In the long-term study, 212 patients received TAKHZYRO 300 mg every 2 weeks for a mean duration of 29.6 (SD=8.2) months. Most patients (81.6%) completed the study or enrolled in commercial product. For rollover patients, the HELP study baseline attack rate (4-week run-in-period) was used as this baseline. For nonrollover patients, baseline was defined as the number of investigator-confirmed attacks reported in the last 3 months. Rollover patients, regardless of randomization group in the pivotal trial, received a single dose of TAKHZYRO 300 mg at study entry and were followed until the first HAE attack occurred. After the first HAE attack, all rollover patients received open-label treatment with TAKHZYRO 300 mg every 2 weeks. Nonrollover patients received open-label treatment with TAKHZYRO 300 mg starting on Day 0 and continued to receive TAKHZYRO every 2 weeks throughout the duration of the treatment period. The long-term safety of TAKHZYRO was the primary endpoint in the study.2
*Androgens or antifibrinolytics. SD=standard deviation
Data were consistent in the open-label extension study1,2
Patients taking TAKHZYRO for an average of 30 months experienced attack reduction vs baseline2
in Attacks
attack rate
attack rate
- 84% reduction in moderate or severe attacks (N=209)2
- 93% reduction in attacks requiring acute treatment (n=106)2
All data presented are for TAKHZYRO 300 mg every 2 weeks unless otherwise indicated.
SELECT IMPORTANT SAFETY INFORMATION
The most commonly observed adverse reactions (≥10% and higher than placebo) associated with TAKHZYRO were injection site reactions; upper respiratory infection; headache; rash; myalgia; dizziness; and diarrhea.
Many patients had zero attacks for extended periods of time when taking TAKHZYRO for an average of 30 months2
On average, patients† experiencedZero Attacks for
PATIENTS (81.8%)
FOR AT LEAST A
6-MONTH PERIOD
(N=209)2
OF DAYS
ON AVERAGE DURING
treatment period
(N=209, SD=6%)2
- 14.8 months was the mean duration of the attack-free period (415 days, SD=12.4 months) (N=209)2
- Mean study duration: 29.6 months (SD=8.2)
All data presented are for TAKHZYRO 300 mg every 2 weeks unless otherwise indicated.
†Included rollover and nonrollover patients
SD=standard deviation.
Long-term data were consistent with the safety and efficacy in the pivotal trial.1,2
View a real patient case from the perspective of the treating HCP
Marie's frequent attacks led her to inquire about preventive treatment.
Continue exploring TAKHZYRO
See safety information established in the pivotal trial and open-label extension study.
