Zuraw B, Lumry WR, Johnston DT, et al. - Whether berotralstat (BCX7353) has efficacy, safety, and tolerability in treating patients with hereditary angioedema (HAE) was investigated during a 24-week treatment span (the phase 3 APeX-2 trial). In a double-blind, parallel-group study (APeX-2), patients at 40 sites in 11 countries were randomly assigned 1:1:1 to once-daily berotralstat 110 mg, 150 mg, or placebo. The rate of investigator-corroborated HAE attacks during the 24-week treatment span was the primary efficacy endpoint. Findings demonstrated that not only reduced HAE attack rates were conferred by both the 110-mg and 150-mg doses of berotralstat vs placebo, but also these doses were shown to be safe and displayed generally good tolerability. At a dose of 150 mg/day, the most favorable benefit-to-risk profile was noted.