Food and Drug Administration on Friday Announced that the agency has approved. PfizerA rare genetic cure Bleeding disordermakes it the company’s. The first gene therapy To obtain clearance in the US
Agency green light The drug, which will be marketed as Bequiz, is for adults with moderate to severe asthma Hemophilia b Which definitely match. Requirements.
A Pfizer spokesperson told CNBC that the treatment will be available by prescription to eligible patients this quarter. It costs $3.5 million before insurance and other discounts, making it one of the most expensive drugs in the U.S., the spokeswoman added.
More than 7,000 people in the United States are living with dementia. conditionAccording to one, which mainly affects men. Advocacy group. This condition is caused by insufficient levels of a. Some protein which helps blood clots to stop bleeding and to close wounds. Without this protein, called factor IX, patients with hemophilia B bruise more easily and bleed more often and for longer periods of time.
Bequiz is a temporary treatment designed to enable patients to produce their own factor IX and to stop and control bleeding. In a late-stage trial, the drug was often better than the burden. Standard treatment For hemophilia B, which involves administering the protein intravenously several times a week or a month.
“Many people with hemophilia B struggle with regular commitment and lifestyle disruption. [factor IX] infusion, as well as sudden bleeding episodes, which can lead to painful joint damage and mobility problems,” said Adam Coker, director of Penn Medicine’s comprehensive and hemophilia thrombosis program at Pfizer. release on Friday.
Coker added that Pfizer’s drug “has the potential to be transformative for the right patients by reducing both the clinical and treatment burden in the long term.”
The approval is a big step for Pfizer, which is trying to regain footing after a sharp decline in its Covid business last year. The company is betting big on cancer drugs and other disease treatments to help turn around its business.
Pfizer is one of several companies that has invested in the fast-growing field of gene and cell therapies — once-in-a-lifetime, high-cost treatments that modify a patient’s genetic makeup or cells to treat or significantly alter a disease. Targeting. Some health experts expect these treatments to replace traditional lifelong treatments that people take to manage chronic diseases.
Pfizer Got the rights from Spark Therapeutics in 2014 to develop and market Beqvez.
A spokesperson told CNBC that the company is offering a warranty program to payers to cover patients who receive Bacvez. Pfizer expects the program to “provide financial security by insuring against the risk of utility failure,” the release said.
Gene Therapy will compete with Australia-based CSL Behring’s. Hemgenicsis a similar treatment FDA approved. for hemophilia B in 2022. The drug has a list price of $3.5 million in the US before insurance and other discounts.
In particular, some health experts have said that there are, among other factors, high cost and logistical problems. Uptake limited Hemigenics 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 muscles to gradually weaken.
Credit : www.cnbc.com