According to a story from BioPharma Dive, the US Food and Drug Administration (FDA) has recently approved Beqvez, a gene therapy from pharmaceutical giant Pfizer intended to treat hemophilia B. It’s approved for adults with moderate to severe symptoms who are currently using other medications to prevent bleeding episodes or who otherwise struggle with repetitive, spontaneous bleeds.
A New Gene Therapy
Before receiving this treatment, eligible patients must also receive testing for an antibody that is known to counteract Beqvez’s effects. With a list price of $3.5 million, this cost matches that of the other approved hemophilia B gene therapy on the market, Hemgenix. While this new approval could very well be a cause for excitement for some patients, the impacts of existing gene therapies for hemophilia have been somewhat underwhelming.
Along with Hemgenix, introduced by UniQure and CSL, BioMarin’s gene therapy for hemophilia A, called Roctavian, has also been on the market for some time. They have demonstrated immense potential in trials, appearing capable of eliminating bleeding for several years. However, relatively few patients have opted for gene therapy.
With several different treatment options now available, it could be that many eligible patients are content with their current medicines and balk when faced with the price and potential hesitation on the part of payers.
Beqvez has also been approved in Canada and is also under evaluation for approval in the EU market.
About Hemophilia
Hemophilia is a genetic disorder which affects the ability of the blood to form clots, a process that is vital for stopping bleeding after a wound is sustained. The severity of symptoms can vary widely. The disorder is caused by a mutation found on the X chromosome. Symptoms include bleeding for a long time after an injury, risk of bleeding in the brain and joints, and easy bruising. Bleeding in the joints can cause permanent damage and brain bleeding can lead to headaches, decreased consciousness, and seizures. There are multiple types of hemophilia, with the most common types being type A and type B, which are distinguished by having deficiencies in different clotting factors. Treatment involves replacing the missing clotting factor. Drugs that thin the blood should be avoided. To learn more about hemophilia, click here.
