UPDATE 2-US FDA approves Pfizer's gene therapy for rare bleeding disorder [Yahoo! Finance]
Pfizer, Inc. (PFE)
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Source: Yahoo! Finance
Food and Drug Administration approved Pfizer's gene therapy for hemophilia B on Friday, 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. The therapy will be available by prescription to eligible patients this quarter, a company spokesperson said. Pfizer has set a list price for Beqvez at $3.5 million in the U.S. - the same price tag as Australian drugmaker CSL Ltd's rival gene therapy Hemgenix. Gene therapies are often pricey, and the durability of a single-d
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