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

Abstract

The 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.

• Authors: Michihiro Hide, Isao Ohsawa, Christina Nurse, Ming Yu, SHP643-302 Trial Investigators
• Journal: The Journal of dermatology (J Dermatol) Vol. 50 Issue 11 Pg. 1381-1391 (Nov 2023) ISSN: 1346-8138 [Electronic] England
• PMID: 37574953 (Publication Type: Clinical Trial, Phase III, Journal Article, Multicenter Study)
• Copyright: © 2023 Takeda Pharmaceuticals and The Authors. The Journal of Dermatology published by John Wiley & Sons Australia, Ltd on behalf of Japanese Dermatological Association.

Chemical References

• Antibodies, Monoclonal, Humanized
• Complement C1 Inhibitor Protein
• lanadelumab

Topics

• Humans
• Angioedemas, Hereditary (drug therapy)
• Antibodies, Monoclonal, Humanized (adverse effects, therapeutic use)
• Complement C1 Inhibitor Protein
• East Asian People
• Injection Site Reaction
• Treatment Outcome
• Adult
• Middle Aged