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The Food and Drug Administration approved Friday Pfizertreatment of a rare genetic bleeding disorder, making it the company’s first-ever gene therapy to gain approval in the United States.
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. Its price is high, $3.5 million, before insurance and other discounts, the spokesperson added, making it by far one of the most expensive drugs in the United States.
More than 7,000 people in the United States live with the debilitating disease, which primarily affects men, according to an advocacy group. The disease is caused by insufficient levels of a certain protein that helps the blood form clots to stop bleeding and seal wounds. Without this 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 enable patients to produce factor IX themselves and prevent and control bleeding. In a late-stage trial, the drug was found to be superior to the often tedious standard treatment for hemophilia B, which involves administering the protein several times a week or month through the veins.
“Many people with hemophilia B have to deal with the commitment and lifestyle disruption of regular (factor IX) infusions, as well as episodes of spontaneous bleeding, which can lead to painful joint damage and mobility issues,” said Dr. Adam Cuker, director of Penn’s Comprehensive and Hemophilia Thrombosis Program, in a Pfizer statement released Friday.
Pfizer’s drug “has the potential to be transformative for affected patients by reducing both the medical and therapeutic burden over the long term,” Cuker added.
The approval is an important 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 illnesses to help turn around its business.
Pfizer is one of several companies investing in the rapidly growing field of gene and cell therapies. These are unique and 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 take to manage chronic illnesses.
Pfizer obtained the production and marketing rights to 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 protections by insuring against the risk of efficacy failure,” they added.
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 United States, before insurance and other discounts.
In particular, some health experts have said that high costs and logistical problems, among other factors, have limited the use of Hemgenix and another gene therapy approved for hemophilia A, which is more common.
Pfizer is also seeking FDA approval for its investigational antibody, marstacimab, to treat hemophilia A and B. The company is also developing a gene therapy for Duchenne muscular dystrophy, a genetic disease that causes progressive weakening muscles.
cnbc
