The Food and Drug Administration announced Friday that the agency had granted approval Pfizeris the treatment of a rare genetic blood clotting disorder, making it the company’s first gene therapy to receive approval in the United States
The agency greenlit the drug, 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. The spokesperson added that the price is a hefty $3.5 million (before insurance and other discounts), making it one of the most expensive drugs in the United States
According to an advocacy group, more than 7,000 people in the United States live with the debilitating disease, which predominantly affects men. The condition is caused by insufficient levels of a certain protein that helps blood clot, stops bleeding and seals wounds. Without this protein, called factor IX, patients with hemophilia B bruise more 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 study, the drug was found to be superior to the often cumbersome standard treatment for hemophilia B, in which the protein is administered through the veins several times a week or month.
“Many people with hemophilia B struggle with commitment and disruption to their lifestyle [factor IX] infusions as well as spontaneous bleeding episodes that can lead to painful joint damage and mobility problems,” Adam Cuker, director of Penn Medicine’s Comprehensive and Hemophilia Thrombosis Program, said in a Pfizer news release Friday.
Pfizer’s drug “has the potential to be transformative for eligible 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 footing after the rapid decline in its Covid business last year. The company is betting heavily on cancer drugs and treatments for other disease areas to turn around its business.
Pfizer is one of several companies investing in the fast-growing field of gene and cell therapies — one-time, expensive treatments that target a patient’s genetic source or cell to cure or significantly alter the course of a disease. Some health experts expect these therapies to replace traditional lifelong treatments that people take to treat chronic illnesses.
Pfizer received the rights to produce 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,” the press release states.
The gene therapy will compete with Australia-based CSL Behring’s Hemgenix, a similar drug that received FDA approval for hemophilia B in 2022. This drug has a similar list price in the US of $3.5 million before insurance and other discounts.
In particular, some health experts have said that high costs and logistical problems, among other things, have limited the rollout 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 disorder that causes the gradual weakening of muscles.
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2024-04-26 13:22:54
www.cnbc.com
