Efficacy and safety of lanadelumab in Japanese patients with hereditary angioedema: A phase 3 multicenter, open‐label study

Abstract

AbstractThe safety and efficacy of lanadelumab for the prevention of hereditary angioedema (HAE) attacks have not been studied in Japanese patients. We report outcomes from a phase 3, multicenter, open‐label study (NCT04180163) of lanadelumab in Japanese patients with HAE. Japanese patients with HAE aged ≥12 years with ≥1 investigator‐confirmed HAE attack during the 4‐week run‐in baseline period were enrolled into the study and received lanadelumab 300 mg every 2 weeks subcutaneously for 52 weeks. Dosing could be reduced to 300 mg every 4 weeks during the second 26‐week treatment period if patients had well‐controlled symptoms (e.g., attack‐free) for 6 months. The primary efficacy endpoint was no investigator‐confirmed HAE attacks (attack‐free status) during days 0–182. Other outcomes included the rate of investigator‐confirmed HAE attacks per month (28 days) and lanadelumab safety. Twelve patients (mean ± SD age 41.9 ± 12.4 years) were enrolled. During the first 26 weeks (days 0–182), five (41.7%) patients were attack‐free. The mean ± SD HAE attack rate per month decreased by 74.0%, from 3.8 ± 2.4 during baseline to 1.2 ± 2.6 during the overall 52‐week treatment period. There were no deaths or discontinuations due to treatment‐emergent adverse events (TEAEs), no severe or serious TEAEs related to lanadelumab, and no positive anti‐drug antibody results. The most frequent TEAEs were injection‐site reactions (37 events in six patients). Most of the injection‐site reaction adverse events were mild in severity. Results of this study support the findings from two global phase 3 studies for lanadelumab use as prophylactic therapy in Japanese patients with HAE.