Background Lanadelumab is a plasma kallikrein inhibitor approved in many countries, including China, for the long-term prophylaxis of hereditary angioedema (HAE). The primary objective was safety evaluation of lanadelumab for HAE long-term prophylaxis in a Chinese patient population. Secondary objectives included efficacy, pharmacokinetics, pharmacodynamics, and immunogenicity evaluations. Methods This open-label, prospective, phase 3, post-approval commitment study included patients in China aged ≥12 years with HAE due to C1 inhibitor deficiency (HAE-C1INH) (NCT05460325). Eligible patients with ≥1 HAE attack in the 4-week run-in period received subcutaneous lanadelumab 300 mg every 2 weeks during days 0–182 for 14 doses in total. Results Twenty patients were enrolled (median age, 38 years; 7 male), and all completed the study. No treatment-emergent adverse events (TEAEs) were fatal, led to treatment discontinuation, or were of special interest. Fifteen (75%) patients reported ≥1 TEAE during the study, most commonly COVID-19 infection (50%). Nine (45%) patients reported treatment-related TEAEs, most frequently injection site pain (20%) and swelling (15%). Injection site reactions occurred in 25% of patients. Mean ± SD monthly rate of investigator-confirmed attacks decreased by 99.1% during days 0–182 versus run-in (0.04 ± 0.14 vs 2.50 ± 1.44). Eighteen (90%) patients were attack free during days 0–182; 2 patients experienced a total of 4 mild and 1 moderate investigator-confirmed HAE attacks. Lanadelumab pharmacokinetics, pharmacodynamics, and immunogenicity in Chinese patients were consistent with other patient populations. Conclusion These findings in patients with HAE in China are consistent with the profile of lanadelumab established in patients from other regions, supporting its use as first-line long-term prophylaxis.
Zhi et al. (Wed,) studied this question.