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U.S. Food and Drug Administration on Friday officially recognized PfizerTreatment of rare genetic diseases bleeding disordersmaking it the company’s The first ever gene therapy Win a U.S. License
agent green light The drug will be marketed as Beqvez to treat adults with moderate to severe disease Hemophilia B people who meet certain conditions Require.
A Pfizer spokesperson told CNBC that the treatment will be available by prescription to eligible patients this quarter.The drug costs $3.5 million before insurance and other rebates, making it one of the most expensive drugs in the United States to date, the spokesman added.
More than 7,000 people in the U.S. suffer from this debilitating disease stateThis mainly affects men, according to one study Initiative group.This situation is caused by insufficient a level certain proteins It helps blood form clots to stop bleeding and close wounds. Without this protein, called factor IX, people with hemophilia B bruise easily and bleed more frequently and last longer.
Beqvez is a one-time treatment designed to enable patients to produce their own Factor IX and prevent and control bleeding.In late-stage trial, drug outperforms often troublesome drugs standard treatment For hemophilia B, protein is given intravenously several times a week or monthly.
“Many hemophilia B patients struggle with the commitment and lifestyle disruption of regular (Factor IX) infusions, as well as spontaneous bleeding, which can lead to painful joints,” said Dr. Adam Cuker, director of Penn General Hospital. Impairments and mobility issues. release on Friday.
Cook added that Pfizer’s drug “has the potential to be transformative for the right patients by reducing long-term medical and treatment burdens.”
The approval is a big step for Pfizer, which is trying to regain its footing after a sharp decline in business last year due to the coronavirus pandemic. The company is investing heavily in cancer drugs and treatments in other disease areas to help turn around its business.
Pfizer is one of several companies investing in the fast-growing field of gene and cell therapies. They are one-time, high-cost treatments that target a patient’s genetic source or cells to cure or significantly alter 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 gained the right Beqvez is manufactured and sold by Spark Therapeutics in 2014.
A spokesperson told CNBC that the company is offering warranty plans to payers to cover patients treated with Beqvez. Pfizer expects the program will “provide financial protection by ensuring the risk of efficacy failure,” they added.
The gene therapy will compete with Australian company CSL Behring Hermogenixsimilar processing Obtain FDA approval Treating Hemophilia B in 2022.
Notably, some health experts say factors such as high costs and logistical issues have led to limited absorption Hemgenix and another approved gene therapy for the more common form of hemophilia A.
Pfizer is also seeking FDA approval for its experimental antibody marstacimab to treat hemophilia A and B. A genetic disorder in which muscles gradually weaken.
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