The Food and Drug Administration on Friday approved Pfizertreatment for a rare genetic disease bleeding disorderbecoming the company first gene therapy to obtain authorization in the USA
The agency green light the drug, which will be marketed as Beqvez, for adults with moderate to severe disease hemophilia B that meet certain requirements.
The treatment will be available by prescription to eligible patients this quarter, a Pfizer spokesperson told CNBC. It has a hefty price tag of $3.5 million before insurance and other discounts, the spokesperson added, making it by far one of the most expensive drugs in the US.
More than 7,000 people in the US live with the debilitating disease illnesswhich predominantly affects men, according to a study Advocacy group. The condition is caused by insufficient levels of a certain protein which helps 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 allow patients to produce factor IX themselves and prevent and control bleeding. In a late-stage trial, the drug was superior to the often complicated drug standard treatment for hemophilia B, which involves administering the protein several times a week or a month through the veins.
“Many people with hemophilia B struggle with compromising and interrupting their lifestyle. [factor IX] infusions, as well as spontaneous bleeding episodes, which can cause painful joint damage and mobility problems,” said Dr. Adam Cuker, director of the Penn Hemophilia Comprehensive and Thrombosis Program, in a Pfizer study. release on Friday.
Pfizer’s drug “has the potential to be transformative for appropriate patients, 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 transform your business.
Pfizer is one of several companies investing in the fast-growing field of gene and cell therapies. These are unique, high-cost treatments that target a patient’s genetic source or cell to cure or significantly alter the course of a disease. Some health experts hope cell and gene therapies will replace the traditional, lifelong treatments people take to manage chronic diseases.
Pfizer won the rights to produce and commercialize Beqvez from Spark Therapeutics in 2014.
The company is offering payers a guarantee program to cover patients who receive Beqvez, a spokesperson told CNBC. Pfizer expects this program to offer “financial protections, ensuring against the risk of efficacy failure,” they added.
The gene therapy will compete with Australia-based CSL Behring Hemgenixa similar treatment that gained FDA approval for hemophilia B in 2022. That drug has a similar list price of $3.5 million in the U.S., before insurance and other discounts.
Notably, some healthcare experts have stated that high costs and logistical issues, among other factors, limited absorption of Hemgenix and another approved gene therapy for the most 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 disease that causes gradual weakening of muscles.
This article was originally published in NBCNews. with
