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Pfizer’s First Gene Therapy for Rare Inherited Bleeding Disorder Receives FDA Approval

Pfizer has received FDA approval for its first-ever gene therapy for a rare genetic bleeding disorder, marking a significant milestone for the company. The treatment, called Beqvez, is designed for adults with moderate to severe hemophilia B who meet certain requirements. This one-time treatment aims to enable patients to produce a specific protein called factor IX, which helps blood form clots and stop bleeding. Hemophilia B patients often struggle with regular infusions of factor IX and experience painful joint damage and mobility issues due to spontaneous bleeding episodes.

The approval of Beqvez is a major breakthrough for Pfizer as it seeks to rebound from the decline of its Covid business last year. The company is focusing on cancer drugs and treatments for other disease areas to revitalize its business. Additionally, Pfizer is among several companies investing in gene and cell therapies, which are high-cost treatments that target a patient’s genetic source or cell to cure or significantly alter the course of a disease. Experts predict that these therapies may replace traditional lifelong treatments for managing chronic diseases.

Pfizer acquired the rights to produce and market Beqvez from Spark Therapeutics in 2014. The drug is expected to be available to eligible patients this quarter, although it comes with a hefty price tag of $3.5 million before insurance and rebates. This makes Beqvez one of the most expensive drugs in the U.S. However, Pfizer is offering a warranty program to provide financial protection against efficacy failure for patients receiving Beqvez.

Beqvez will face competition from CSL Behring’s Hemgenix, another gene therapy treatment for hemophilia B that received FDA approval in 2022. Hemgenix also carries a price tag of $3.5 million before insurance and rebates. However, experts have noted that high costs and logistical issues have limited the uptake of gene therapies for hemophilia A and B.

In addition to Beqvez, Pfizer is seeking 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 progressive weakening of muscles.

The approval of Beqvez represents a significant advancement in the treatment of rare genetic bleeding disorders. By providing a one-time treatment option that enables patients to produce the necessary clotting protein, Pfizer’s gene therapy has the potential to reduce the medical and treatment burden for individuals with hemophilia B. With the growing field of gene and cell therapies, the future of managing chronic diseases may be transformed, offering new hope for patients and potentially replacing lifelong treatments.

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