The Food and Drug Administration announced on Friday the approval of Pfizer’s treatment for a rare genetic bleeding disorder, marking the company’s first gene therapy to receive clearance in the U.S.
The agency approved the drug, which will be marketed as Beqvez, for adults with moderate to severe hemophilia B who meet certain criteria.
The treatment will be available by prescription to eligible patients this quarter, according to a Pfizer spokesperson who told CNBC. With a price tag of $3.5 million before insurance and rebates, Beqvez is among the most expensive drugs in the U.S.
More than 7,000 people in the U.S. suffer from this debilitating condition, which predominantly affects men, according to an advocacy group.
The disorder is caused by insufficient levels of a protein that helps blood clot to stop bleeding and seal wounds.
Without this protein, called factor IX, patients with hemophilia B bruise easily and experience prolonged and more frequent bleeding.
Beqvez is a one-time treatment designed to enable patients to produce factor IX themselves, preventing and controlling bleeding.
In a late-stage trial, Beqvez outperformed the standard treatment for hemophilia B, which involves administering the protein multiple times a week or month through the veins.
“Many people with hemophilia B struggle with the commitment and lifestyle disruption of regular [factor IX] infusions, as well as spontaneous bleeding episodes, which can lead to painful joint damage and mobility issues,” said Adam Cuker, director of Penn Medicine’s Comprehensive and Hemophilia Thrombosis Program, in a Pfizer release on Friday.
Pfizer’s drug “has the potential to be transformative for appropriate patients by reducing both the medical and treatment burden over the long term,” Cuker added.
The approval is a significant milestone for Pfizer, which is trying to regain its footing following the rapid decline of its Covid business last year.
The company is heavily investing in cancer drugs and treatments for other disease areas to rejuvenate its business.
Pfizer is one of several companies investing in the burgeoning field of gene and cell therapies — 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 anticipate these therapies will replace traditional lifelong treatments for chronic diseases.
Pfizer gained the rights to produce and market Beqvez from Spark Therapeutics in 2014.
The company is offering payers a warranty program to cover patients who receive Beqvez, a spokesperson told.
Pfizer expects the program to offer “financial protection by insuring against the risk of efficacy failure,” according to the release.
The gene therapy will compete with Australia-based CSL Behring’s Hemgenix, a similar treatment approved by the FDA for hemophilia B in 2022.
Hemgenix also has a list price of $3.5 million in the U.S. before insurance and rebates.
Notably, some health experts have pointed out that high costs and logistical challenges have limited the uptake 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.
Additionally, the company is developing a gene therapy for Duchenne muscular dystrophy, a genetic disorder that gradually weakens muscles.
