BioCryst Pharmaceuticals, Inc. announced that data from the open-label extension (OLE) of the APeX-2 trial of oral, once-daily ORLADEYO® (berotralstat) for the prophylactic treatment of hereditary angioedema (HAE) in patients 12 years and older have been published online by the Journal of Allergy and Clinical Immunology: In Practice (JACI: In Practice). APeX-2 was a Phase 3, double-blind, placebo-controlled, parallel-group, three-part study evaluating ORLADEYO versus placebo for the prevention of HAE attacks in 121 patients with HAE Type I or Type II. In part 3 of the study (weeks 49-96), all patients (n=81) were treated with open-label ORLADEYO at 150 mg.

The primary endpoint of the OLE was long-term safety and tolerability, while secondary endpoints included HAE attack rates and quality of life (QoL). The authors concluded that ORLADEYO was generally well tolerated, provided rapid and sustained reductions in HAE attacks and improved QoL over the study duration of 96 weeks. The results from part 3 of APeX-2 were first presented at the European Academy of Allergy and Clinical Immunology (EAACI) Congress in July 2021.