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The Food and Drug Administration granted approval on Friday Pfizer‘s treatment for a rare genetic blood disorder, making it the company’s first-ever gene therapy to receive approval in the US
The agency has given the green light to the drug, which will be marketed as Beqvez, for adults with moderate to severe hemophilia B who meet certain requirements.
The treatment will be available by prescription to eligible patients this quarter, a Pfizer spokesperson told CNBC. It carries a hefty price tag of $3.5 million, before insurance and other discounts, the spokesperson added, making it by far one of the most expensive drugs in the US.
More than 7,000 people in the US suffer from the debilitating condition, which mainly affects men, according to an advocacy group. The condition is caused by insufficient amounts of a certain protein that helps the blood form 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, which involves giving the protein 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,” Dr. Adam Cuker, director of Penn’s Comprehensive and Hemophilia Thrombosis Program, said in a Pfizer press release 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 manufacture and market Beqvez from Spark Therapeutics in 2014.
The company offers payers a guarantee program to cover patients who receive Beqvez, a spokesperson told CNBC. Pfizer expects this program to “provide financial protection by insuring against the risk of efficacy failure,” she added.
The gene therapy will compete with CSL Behring’s Australia-based Hemgenix, a similar treatment that won FDA approval for hemophilia B in 2022. That drug has a similar list price of $3.5 million in the U.S., before insurance and other rebates .
In particular, some health experts have said that high costs and logistical issues, among other issues, have limited acceptance of 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.