Lanadelumab safety, efficacy, and pharmacokinetics in patients aged ≥12 with hereditary angioedema in China: an open-label, multicenter study.

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.