$2.9 million gene therapy for severe hemophilia is approved by FDA

By MATTHEW PERRONE
AP Health Writer

WASHINGTON (AP) — U.S. health regulators have approved a gene therapy for the most common form of hemophilia. The treatment, Roctavian, is an infusion that’s been shown to reduce dangerous bleeding problems caused by the inherited condition. The Food and Drug Administration approved it Thursday for adult patients with severe cases of hemophilia A. That’s the blood-clotting disorder that can lead to bleeding after minor injuries or scrapes. Drugmaker BioMarin said it will charge $2.9 million for the therapy. The therapy is a long-awaited alternative to current treatments. Those include weekly doses of a protein needed to help blood clot.