NBC News: FDA approves Pfizer’s first gene therapy for rare inherited blood disorder


On Friday, the Food and Drug Administration greenlit Pfizer’s treatment for adults with moderate to severe hemophilia B. Hemophilia B is a blood condition caused by insufficient amounts of a certain protein needed to form blood clots. Without sufficient levels of the protein called factor IX, those with the condition can bruise easily and bleed more frequently at longer intervals. 

This drug will be marketed as Beqvez and will be available by prescription according to a Pfizer spokesperson. The $3.5 million list price before insurance or other rebates will make this one of the most expensive drugs in the United States. In contrast to the standard treatment of administering the protein multiple times a week, Beqvez is a one-time treatment intending to enable patients to produce factor IX themselves.  
To read the full article by Annika Kim Constantino, click here.

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