The Food and Drug Administration announced Friday that the agency approved Pfizeris the treatment for a rare genetic bleeding disorder, making it the company's first gene therapy to gain clearance in the US.
The agency gave 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 reimbursements, the spokesperson added, making it by far one of the most expensive drugs in the U.S.
According to one advocacy group, more than 7,000 people in the United States live with this debilitating disease, which predominantly affects men. The condition is caused by insufficient levels of a certain protein that helps blood clot, stop bleeding, and seal wounds. Without that protein, called factor IX, patients with hemophilia B bruise easily and bleed more frequently and for longer periods of time.
Beqvez is a unique treatment designed to allow patients to produce factor IX on their own and prevent and control bleeding. In a late-stage trial, the drug was superior to the often cumbersome standard treatment for hemophilia B, which involves administering the protein several times a week or month through the veins.
“Many people with hemophilia B struggle with lifestyle commitment and disruption of their usual activities. [factor IX] infusions, as well as spontaneous bleeding episodes, which can lead to painful joint damage and mobility problems,” Adam Cuker, director of Penn Medicine's Hemophilia Thrombosis and Comprehensive Program, said in a Pfizer statement on Friday.
Pfizer's drug “has the potential to be transformative for appropriate patients by reducing long-term medical and treatment burden,” Cuker added.
The approval is a big step for Pfizer, which is trying to regain its footing 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 several companies investing in the rapidly growing field of gene and cell therapies: unique, high-cost treatments that target a patient's genetic or cellular source to cure or significantly alter the course of a disease. Some health experts hope these therapies will replace the traditional lifelong treatments that people take to manage chronic diseases.
Pfizer obtained the rights to produce and market Beqvez from Spark Therapeutics in 2014.
The company offers payers an assurance program to cover patients receiving Beqvez, a spokesperson told CNBC. Pfizer hopes that program will offer “financial protection by insuring against the risk of efficacy failure,” according to the statement.
The gene therapy will compete with Australia-based CSL Behring's 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. .US before insurance and other reimbursements.
In particular, some health experts have said that high costs and logistical problems, among other factors, have limited the acceptance of Hemgenix and another gene therapy approved 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 muscles to gradually weaken.
