US FDA approves BioMarin’s gene therapy for hemophilia A

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BioMarin’s gene therapy, which would be the first for hemophilia A, is a potentially one-time treatment that would enable patients to forego or reduce infusions of factor proteins into their bloodstream several times a week for life.

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.

BioMarin’s therapy was approved for the same condition in the European Union last year, and is sold under the same brand name.

In April, BioMarin cut its annual sales forecast range for Roctavian to $50 to $100 million, from $100 to $200 million.

The company said on Thursday it expects about 2,500 of around 6,500 adults living with severe hemophilia A in the United States to be eligible to receive Roctavian with this initial approval.

There are about 16,000 patients in the United States with hemophilia A in which they are missing the factor VIII clotting protein.