Long-term safety and effectiveness of berotralstat for hereditary angioedema: The open-label APeX-S study.

ABSTRACT:

Background

Berotralstat (BCX7353) is an oral, once-daily inhibitor of plasma kallikrein recently approved for prevention of angioedema attacks in adults and adolescents with hereditary angioedema (HAE). The objective of this report is to summarize results from an interim analysis of an ongoing long-term safety study of berotralstat in patients with HAE.

Methods

APeX-S is an ongoing, phase 2, open-label study conducted in 22 countries (ClinicalTrials.gov, NCT03472040). Eligible patients with a clinical diagnosis of HAE due to C1 inhibitor deficiency (HAE-C1-INH) were centrally allocated to receive berotralstat 150 or 110 mg once daily. The primary objective was to determine long-term safety and the secondary objective was to evaluate effectiveness.

Results

Enrolled pat

SUBMITTER: Farkas H 

PROVIDER: S-EPMC8221587 | biostudies-literature | 2021 Jun

REPOSITORIES: biostudies-literature