US FDA Approves BioMarin Pharma's gene therapy for hemophilia A

The U.S. Food and Drug Administration later this week 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. It priced the one-time therapy, Roctavian, at $2.9 million. Pivotal trial results showed that Roctavian reduced bleeding events, but its durability is not known, and the firm said it would include a warranty to health insurers. BioMarin said most study participants continued to respond to the gene therapy through year three and beyond, and it would continue to monitor them for 15 years.

Gene therapies use a deactivated virus to deliver the needed genes and the immune system would recognise and eliminate a second dose of the therapy. The firm said it would offer all U.S. health insurers a "warranty", under which it would reimburse the wholesale cost if a patient does not respond. The company said the reimbursement would be "pro-rated" for patients that stopped responding in the first four years after treatment. Shares of the California-based company closed 3.6 per cent lower on later this week. "Roctavian's launch will probably be a little slow at first, because there are already some effective treatment options on the market. But longer-term, one-time dosing will likely end up being attractive to a lot of patients," Beatty added.

Roctavian was approved for hemophilia A 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 million to $100 million, from $100 million to $200 million. There are about 16,000 patients in the United States with hemophilia A in which they are missing the factor VIII clotting protein.