FDA approves Pfizer’s first gene therapy for rare inherited bleeding disorder

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Pfizer gene therapy

Pfizer’s treatment for a rare bleeding disorder receives FDA approval, marking the company’s first gene therapy clearance in the U.S.

The recent FDA approval of Pfizer gene therapy marks a significant milestone in the treatment of rare inherited bleeding disorders.

The agency greenlighted the drug, which will be marketed as Beqvez, for adults with moderate to severe hemophilia B. 

Pfizer plans to make the treatment accessible to eligible patients through prescriptions this quarter. However, its high price tag of $3.5 million may pose challenges for some, even with insurance coverage.

The treatment will be available by prescription to eligible patients this quarter, a Pfizer spokesperson told CNBC. The spokesperson noted its hefty $3.5 million price tag before discounts, making it one of the priciest drugs in the U.S.

“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 Adam Cuker, director of Penn Medicine’s Comprehensive and Hemophilia Thrombosis Program, in a Pfizer release on Friday.

As Pfizer continues to refine and expand its gene therapy portfolio, patients with rare inherited bleeding disorders can look forward to a brighter future with improved treatment options.

In conclusion, Pfizer’s FDA approval for its gene therapy heralds a new era in the treatment of rare inherited bleeding disorders. Pfizer’s approval marks a milestone for both the company and the scientific field. It also offers renewed optimism to families grappling with such challenging health conditions.
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