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

Health Technology

(FILES) - A file picture taken on October 20, 2009 shows a sign for pharmaceutical company Pfizer Incorporated headquarters in New York. The US pharmaceutical company Pfizer, which plans to further raise its staggering offer to buy its British rival AstraZeneca, is faced with a rising political pressure on both sides of the Atlantic. CEO Ian Read is preparing to fly to Britain where he will be heard on May 13, 2014 by British lawmakers worried the social impact of this potential marriage to more than 100 billion dollars. AFP PHOTO / TIMOTHY A. CLARYTIMOTHY A. CLARY/AFP/Getty Images

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.