Pfizer reported a list price of $3.5 million for its new gene therapy, Beqvez, for adults with this rare bleeding disorder.
Beqvez is the second gene therapy the agency has approved for hemophilia B, a deficiency in clotting factor IX because of a faulty gene that occurs mostly in males. ThePfizer said the list price of Beqvez will be $3.5 million — the same price as Hemgenix. The argument for this hefty price tag is that these gene therapies offer the possibility of a cure whereas ongoing factor IX infusions can cost more than $20 million over a patient's lifetime.
The annualized bleeding rate fell from a mean of 4.5 events during the pretreatment period of at least 6 months to a mean of 2.5 events between week 12 and data cutoff , according to. Overall, bleeding events were eliminated in 60% of patients who received the one-time infusion vs 29% of patients on factor IX prophylaxis therapy.
Overall, Pfizer reported that the gene therapy was"generally well-tolerated," with an increase in transaminases reported as the most common adverse event. No deaths, serious infusion reactions, thrombotic events, or development of factor IX antibodies occurred. Pfizer has said it will continue to monitor patients to assess the therapy's long-term durability and safety over a 15-year period.All material on this website is protected by copyright, Copyright © 1994-2024 by WebMD LLC. This website also contains material copyrighted by 3rd parties.
Haemophilia Biologic Therapy Biologics Genomics Genomic Medicine Gene Therapy Hemophilia B Haemophilia B Hemophilia A Haemophilia A Hemorrhage Bleeding Prophylaxis Aminotransferase Ast Liver Enzymes Liver Studies Alt Transaminase U.S. Food And Drug Administration United States Food And Drug Administration Fda
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