The U.S. Food and Drug Administration on Thursday approved BioMarin Pharmaceutical's gene therapy for severe hemophilia A, the company said, giving patients with the inherited bleeding disorder an alternative to regular injections of missing blood proteins.
gene therapy for severe hemophilia A, the company said, giving patients with the inherited bleeding disorder an alternative to regular injections of missing blood proteins.
Gene therapies use a deactivated virus to deliver the needed genes and the immune system would recognize and eliminate a second dose of the therapy. Roctavian, which is manufactured at the company's facility in Novato, California, works by delivering a functional copy of the missing gene that would help hemophilia A patients make a blood-clotting protein known as factor VIII.
In April, BioMarin cut its annual sales forecast range for Roctavian to $50 million to $100 million, from $100 million to $200 million.
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