FDA Approves Pfizer Gene Therapy Beqvez for Treating Hemophilia B

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The Food and Consumer Product Safety Authority on Friday approved Pfizer's treatment for a rare genetic disorder bleeding disordermaking it the company's very first gene therapy to get permission in the US

The agency illuminated green the drug, which will be marketed as Beqvez, for adults with moderate to severe symptoms hemophilia B meet that certain one requirements.

More than 7,000 people in the US live with debilitating diseases conditionaccording to a advocacy group. The condition is caused by insufficient levels of a certain protein that helps form blood clots to stop bleeding and close wounds. Without that protein, called factor IX, patients with hemophilia B bruise easily and bleed more often and for longer.

Beqvez is a one-time treatment designed to enable patients to produce factor IX themselves and prevent and control bleeding. In a late-stage study, the drug was superior to the often cumbersome standard treatment for hemophilia B, where the protein is administered through the veins several times a week or a month.

“Many people with hemophilia B struggle with the commitment and disruption of regular lifestyles [factor IX] infusions, as well as spontaneous bleeding, which can lead to painful joint damage and mobility problems,” says Dr. Adam Cuker, director of Penn's Comprehensive and Hemophilia Thrombosis Program, in a Pfizer report. Edition on Friday.

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

The approval is a big step for Pfizer, which is trying to regain its position after the rapid decline of its Covid business last year. The company is betting big on cancer drugs and treatments for other disease areas to help improve its business.

Pfizer is one of many companies investing in the fast-growing field of gene and cell therapies. They are one-time, expensive treatments that target a patient's genetic source or cell to cure or significantly change the course of a disease. Some health experts expect cell and gene therapies to replace the traditional lifelong treatments people use to manage chronic diseases.

Pfizer acquired the rights to produce and commercialize Spark Therapeutics' Beqvez in 2014. The company has not disclosed how it will price the treatment.

The gene therapy will compete with Australia-based CSL Behring Hemgenixsimilar treatment as received FDA approval for hemophilia B in 2022. In the US, that drug has a hefty list price of $3.5 million, excluding insurance and other discounts.

In particular, some health experts have said that this comes with high costs and logistical problems, among other things limited uptake from Hemgenix and another approved gene therapy for the more common hemophilia A.

Pfizer is also 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 condition that causes muscles to gradually weaken.

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