FDA approves Pfizer's first gene therapy for rare inherited bleeding disorder [MSNBC.com]
Pfizer, Inc. (PFE)
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Source: MSNBC.com
The Food and Drug Administrationon FridayPfizerbleeding disorderfirst-ever gene therapyThe agencygreenlithemophilia BrequirementsThe treatment will be available by prescription to eligible patients this quarter, a Pfizer spokesperson told . It has a hefty $3.5 million price tag, before insurance and other rebates, the spokesperson added, making it by far one of the most expensive drugs in the U.S.More than 7,000 people in the U.S. are living with the debilitatingconditionadvocacy groupcertain proteinBeqvez is a one-time treatment designed to enable patients to produce factor IX themselves and prevent and control bleeding. In a late-stage trial, the drug was superior to the often-cumbersomestandard treatment“Many people with hemophilia B struggle with the commitment and lifestyle disruption of regular [factor IX] infusions, as well as spontaneous bleeding episodes, which can lead to painful joint damage and mobility issues,” said Dr. Adam Cuker, director of Penn’s Comprehensive and Hem
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