A long-term, 3-year study with real-world evidence in 109 patients3
Study design4
The primary objective of this study was to evaluate the real-world effectiveness of TAKHZYRO as measured by the HAE attack rate before and after TAKHZYRO initiation in patients aged 12 years and older.3
*Established patients were defined as those who had received ≥4 doses of TAKHZYRO before enrollment and were either receiving TAKHZYRO at enrollment or received their most recent dose <70 days before enrollment.3
†New patients were defined as those who had not started TAKHZYRO at enrollment or started TAKHZYRO before enrollment but received <4 doses.3
Limitations of EMPOWER3,4
- This was an observational, self-controlled, real-world study lacking a standardized treatment protocol or control arm3,4
- Small sample size due to the rarity of HAE3,4
- Potential selection bias due to including established patients3,4
- Possible misclassification of effectiveness and safety data due to patient HAE attack diary data capture and recall bias3,4
- Follow-up duration was truncated in some patients due to the sponsor ending the study earlier than planned, which may have resulted in conservative estimates of patients persisting with long-term treatment3,4
- New patients were defined as those who had not started TAKHZYRO at enrollment or started TAKHZYRO before enrollment but received <4 doses. 1 new patient received 1 dose of TAKHZYRO before study enrollment; another new patient had 3 doses of TAKHZYRO before study enrollment3,4
Statistical analysis for EMPOWER:
To assess the effectiveness of TAKHZYRO in new patients, mean attack rate (attacks/month), incidence rate ratios (IRRs), and 95% confidence intervals (CIs) were calculated by a generalized linear model for pre-TAKHZYRO, early-, and steady-state periods. To assess the effectiveness of TAKHZYRO in established patients, mean attack rate and 95% CIs were calculated for the overall study period.
HAE attack reduction seen in real-world evidence for up to 32 months
Reductions in mean monthly HAE attack rates3
The limitations of this noninterventional study include lack of a standardized treatment protocol and a control arm, with most safety and effectiveness parameters based on participants’ recall or self-reported information.
Some patients in the study reported concomitant use of other medications for STP or for LTP while transitioning to TAKHZYRO.
Definition of incidence rate ratio (IRR): a measure to compare the pre-TAKHZYRO attack rate and the on-TAKHZYRO attack rate over a specific period of time. Percent reduction was calculated by: (1-IRR) x 100.
*Early state was from TAKHZYRO initiation to Day 69 and steady state was from Day 70 after TAKHZYRO initiation.
CI=confidence interval; IRR=incidence rate ratio; LTP=long-term prophylaxis; STP=short-term prophylaxis.
Patient times on treatment4
- 32 months (SD=17) in established TAKHZYRO patients (n=91)
- 26 months (SD=13) in new TAKHZYRO patients (n=18)
SELECT IMPORTANT SAFETY INFORMATION
Adverse Reactions: The most commonly observed adverse reactions (≥10%) associated with TAKHZYRO were injection site reactions consisting mainly of pain, erythema, and bruising at the injection site; upper respiratory infection; headache; rash; dizziness; diarrhea; and myalgia. Less common adverse reactions observed included elevated levels of transaminases; one patient discontinued the trial for elevated transaminases.
Reduced HAE attack rates observed in dosing subgroups
Mean attack rates per month in new and established TAKHZYRO patients in the full analysis set3*
The recommended starting dose in patients 12 years of age and older is 300 mg administered subcutaneously every 2 weeks. A dosing interval of 300 mg every 4 weeks may be considered if the patient is well controlled (eg, attack free) for more than 6 months.1
*Attack rates were calculated based on patients with available data in the FAS.
†Early state was from TAKHZYRO initiation to Day 69 and steady state was from Day 70 after TAKHZYRO initiation.
FAS=full analysis set; Q2W=every 2 weeks; Q4W= every four weeks.
References: 1. Takhzyro. Prescribing information. Dyax Corp; 2025. 2. Banerji A, Riedl MA, Bernstein JA, et al. JAMA. 2018;320(20):2108-2121. doi:10.1001/jama.2018.16773 3. Data on File. EMPOWER Study. 4. Bernstein JA, Betschel SD, Busse PJ, et al. Adv Ther. 2025;42(8):3882-3901. doi:10.1007/s12325-025-03226-3