Based upon the COMP positive opinion, the commission will make a final decision on European orphan drug designation.
BCX4161 is a selective inhibitor of plasma kallikrein and is being developed for prevention of attacks in patients with HAE. It was granted orphan drug designation by the US Food and Drug Administration (FDA) in December 2014.
The company said that by inhibiting plasma kallikrein, BCX4161 suppresses production of bradykinin, which is the mediator of acute swelling attacks in HAE patients.
In May 2014, the company reported positive results from the Oral ProphylaxiS-1 (OPuS-1) proof of concept Phase IIa trial of orally-administered BCX4161 in HAE patients.
Primary efficacy endpoint, several secondary endpoints and all other established objectives, were all met in the trial.
The trial’s primary efficacy endpoint was the by-subject difference in mean angioedema attack rate on BCX4161 compared to placebo.
According to the company, treatment with BCX4161 showed a statistically significant mean attack rate reduction of 0.45 attacks per patient-week versus placebo.