#BCRX "announced that data from the first 24 weeks of the APeX-2 trial of oral, once-daily berotralstat in patients with hereditary angioedema (HAE) have been published online by the Journal of Allergy and Clinical Immunology (JACI).
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 U.S. Food and Drug Administration (FDA) is reviewing a new drug application (NDA) for ORLADEYO™ (berotralstat) and has set an action date of December 3, 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" (see press release HERE).
See our BCRX AmpCard to learn more.