Oral berotralstat for the prophylaxis of hereditary angioedema attacks in patients in Japan: A phase 3 randomized trial

Autor: Eniko Nagy, Yoshihiro Sasaki, William P. Sheridan, Michihiro Hide, Masaki Tago, Daisuke Honda, Tomoo Fukuda, Isao Ohsawa, Melanie Cornpropst, Sylvia Dobo, Yusuke Suzuki, Lacy Reese, Greg Chittick, Phil Collis, Jessica M. Best, S. Murray, Shinichi Moriwaki, Osamu Ishikawa, Sharon Van Dyke, Heather Iocca, Keisuke Kohga, Eishin Morita
Rok vydání: 2020
Předmět:
Zdroj: Allergy
ISSN: 1398-9995
0105-4538
Popis: Background With no approved treatments in Japan for the prevention of hereditary angioedema (HAE) attacks, there is a significant unmet need for long‐term prophylactic therapies for Japanese patients with HAE. Berotralstat (BCX7353) is an oral, once‐daily, highly selective inhibitor of plasma kallikrein in development for prophylaxis of angioedema attacks in HAE patients. Methods APeX‐J is a phase 3, randomized, double‐blind, placebo‐controlled, parallel‐group, 3‐part trial conducted in Japan (University Hospital Medical Information Network identifier, UMIN000034869; ClinicalTrials.gov identifier, NCT03873116). Patients with a clinical diagnosis of type 1 or 2 HAE underwent a prospective run‐in period of 56 days to determine eligibility, allowing enrollment of those with ≥2 expert‐confirmed angioedema attacks. Patients were randomly assigned (1:1:1) and stratified by baseline attack rate (≥2 vs.
APeX‐J is a phase 3, placebo‐controlled trial conducted in Japan to assess the efficacy and safety of oral berotralstat in patients with HAE. Patients were randomized 1:1:1 to receive once‐daily berotralstat 110 mg, berotralstat 150 mg, or placebo. Berotralstat 150 mg significantly reduced the frequency of HAE attacks compared with placebo, supporting its use as a prophylactic therapy. Abbreviation: HAE, hereditary angioedema.
Databáze: OpenAIRE
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