FDA approves Pfizer gene therapy Beqvez for treatment of hemophilia B

[Pelican Press 0]

FDA approves Pfizer gene therapy Beqvez for treatment of hemophilia B

Pavlo Gonchar | Lightrocket | Getty Images

The Food and ***** Administration

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approved Pfizer‘s treatment for a rare genetic

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, making it the company’s

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to win clearance in the U.S. 

The agency

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the *****, which will be marketed as Beqvez, for adults with moderate to severe

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who meet certain

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.

More than 7,000 people in the U.S. are living with the debilitating

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, according to an

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. The condition is caused by insufficient levels of a

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that helps blood form clots to stop bleeding and seal wounds. Without that protein, called factor IX, patients with hemophilia B bruise easily and bleed more frequently and for longer periods of time. 

Beqvez 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 ***** was superior to the often-cumbersome

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for hemophilia B, which involves administering the protein multiple times a week or a month through the veins. 

“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 ****** damage and mobility issues,” said Dr. Adam Cuker, director of Penn’s Comprehensive and Hemophilia Thrombosis Program, in a Pfizer

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on Friday.

Pfizer’s ***** “has the potential to be transformative for appropriate patients by reducing both the medical and treatment burden over the long term,” Cuker added. 

The approval is a big step for Pfizer, which is trying to regain its footing following the rapid decline of its Covid business last year. The company is ******** big on ******* drugs and treatments for other ******** areas to help turn its business around. 

Pfizer is one of several companies to invest in the rapidly growing field of gene and cell therapies. They are one-time, high-cost treatments that target a patient’s genetic source or cell to cure or significantly alter the course of a ********. Some health experts expect cell and gene therapies to replace traditional lifelong treatments that people take to manage chronic *********. 

Pfizer

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to produce and market Beqvez from Spark Therapeutics in 2014. The company did not disclose how it will price the treatment.

The gene therapy will compete with Australia-based CSL Behring’s

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, a similar treatment that

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for hemophilia B in 2022. In the U.S., that ***** has a hefty list price of $3.5 million before insurance and other rebates. 

Notably, some health experts have said that high costs and logistical issues, among other factors, have

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of Hemgenix and another approved gene therapy for the more common hemophilia A. 

Pfizer also seeks FDA approval for its experimental antibody, marstacimab, to treat hemophilia A and B. The company is also developing a gene therapy for Duchenne muscular dystrophy, a genetic disorder that causes muscles to weaken gradually. 

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Breaking news,Health care industry,Pharmaceuticals,Biotech and Pharmaceuticals,Biotechnology,Business,Pfizer Inc,Science,******* States,business news
#FDA #approves #Pfizer #gene #therapy #Beqvez #treatment #hemophilia

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