Gene Therapy for Hemophilia A Receives Orphan Designation

The U.S. Food and Drug Administration granted orphan drug designation to BMN 270, an investigational gene therapy for patients with hemophilia A. BMN 270 is an adeno-associated virus 5 factor VIII vector designed to restore factor VIII plasma concentrations in patients with hemophilia A, which is caused by a missing or deficient factor VIII.

A phase I/II, dose-escalating study evaluating the drug’s safety and efficacy in up to 12 patients with severe hemophilia A is underway; primary endpoints of the study include the safety of a single intravenous administration of BMN 270 and the change in factor VIII expression level from baseline to 16 weeks after infusion. Secondary endpoints include the effect of BMN 270 on the frequency of factor VIII replacement therapy, the number of bleeding episodes requiring treatment, and immune responses.

An update on the trial will be available in April, and patients will be monitored for safety and durability for five years.

Source: BioMarin Pharma press release, March 1, 2016.