The Food and Drug Administration approved Pfizer’s treatment of rare genetic diseases and bleeding disorders, making it the company’s first-ever gene therapy. The approval came on Friday. This groundbreaking gene therapy, named Bekvez, is for adults with moderate to severe hemophilia B, meeting specific requirements. Pfizer spokesperson mentioned that the medication will be available by prescription to eligible patients this quarter with a hefty price tag of $3.5 million, making it one of the most expensive drugs in the United States.
More than 7,000 people in the US live with hemophilia B, primarily affecting men as it causes a lack of Factor IX, a crucial protein for blood clotting. Bekvez is designed as a one-time treatment to enable patients to produce their own Factor IX to prevent and control bleeding effectively.
Pfizer’s approval for Bekvez is a major milestone in the company’s efforts to diversify its portfolio after facing challenges due to the decline in its COVID-19 business. The company has heavily invested in anti-cancer drugs and gene and cell therapies, aiming to revolutionize the treatment of chronic diseases. Pfizer also acquired the rights to manufacture and market Bekvez in collaboration with Spark Therapeutics.
In addition, Pfizer is seeking FDA approval for marstasimab, an experimental antibody to treat hemophilia A and B. The company is also developing a gene therapy for Duchenne muscular dystrophy. Bekvez will be competing with similar gene therapies like Hemogenix from CSL Behring, which also comes with a significant price tag of $3.5 million in the US, marking a new era in the treatment of hemophilia.
Despite the high costs and logistical challenges associated with gene therapies, medical experts are hopeful that these one-time treatments can replace traditional lifelong treatments for chronic diseases, offering long-term benefits to patients.
Source: www.nbcnews.com
