The Journal of Allergy and Clinical Immunology (JACI) publishes data from the first 24 weeks of the APeX-2 trial of oral, once-daily berotralstat in patients with HAE.
APeX-2 was a Phase 3, double-blind, placebo-controlled, parallel-group trial that evaluated the efficacy and safety of oral, once-daily berotralstat versus placebo over 24-weeks in 121 HAE patients ages 12 years or older. In the trial, after 24 weeks, both the 110-mg and 150-mg doses of berotralstat significantly reduced HAE attack rates compared with placebo and were safe and well tolerated, with greater attack rate reductions observed for the 150-mg dose.
The authors conclude that the combination of efficacy, safety, and tolerability with convenient oral, once-daily dosing will make berotralstat an important addition to the HAE-C1-INH therapeutic armamentarium, if approved.
“The data from APeX-2 show that berotralstat may provide an important oral option for patients with HAE that could allow them to prevent HAE attacks and reduce their overall burden of therapy,” said Bruce Zuraw, M.D., professor of medicine and chief of the Division of Rheumatology, Allergy and Immunology at the University of California School of Medicine, and principal investigator of the APeX-2 trial.
“We are excited to have our results published in JACI, building on the compelling pivotal and longer-term data presented at scientific congresses in the past year. HAE patients are waiting for an oral, once-daily option to control their disease. As the authors have recognized, providing patients with an effective, oral therapy is a major step towards the goal of enabling them to live a normal life,” said Jon Stonehouse, President and CEO of BioCryst Pharmaceuticals, Inc.
The U.S. Food and Drug Administration (FDA) is reviewing a new drug application (NDA) for ORLADEYO™ (berotralstat) and has set an action date of 3 December 2020, under the Prescription Drug User Fee Act (PDUFA).
In Japan, ORLADEYO is being reviewed under Sakigake designation. The Pharmaceutical and Medical Devices Agency (PMDA) has confirmed their regulatory review schedule and the company expects an approval decision in December 2020.
On 30 March 2020, BioCryst announced that the European Medicines Agency (EMA) had validated its marketing authorization application (MAA) submission for ORLADEYO and begun its formal review of the MAA under the centralized procedure. The company expects an opinion from the Committee for Medicinal Products for Human Use (CHMP) within approximately 12 months from MAA validation.