US FDA approves Pfizer’s gene therapy for rare bleeding disorder

THE US Food and Drug Administration approved Pfizer’s gene therapy for hemophilia B on Friday (Apr 26), the second such therapy for the rare bleeding disorder that typically requires regular infusions of a blood-clotting protein.

People with hemophilia have a fault in a gene that regulates production of proteins called clotting factors, which can cause spontaneous as well as severe bleeding following injuries or surgery. It predominately affects males.

Pfizer’s one-time therapy, branded as Beqvez, is designed to stimulate production of the protein, called factor IX (FIX), by the patient’s own body instead of intravenous infusions of FIX multiple times a week or a month.

A single dose of Pfizer’s therapy was shown to work as well as standard-of-care protein infusions after a year, with bleeding completely eliminated in 60 per cent of patients versus 29 per cent who received infusions.

The therapy will be available by prescription to eligible patients this quarter, a company spokesperson said, adding the list price of the therapy would be US$3.5 million in the US.

Hemophilia B is estimated to affect nearly four in every 100,000 US males, while related disorder hemophilia A is estimated to affect 12 in every 100,000 US males, according to government data. REUTERS