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New Drugs to Market – October Update

The Food and Drug Administration (FDA) approved three novel drug therapies in October. One of those therapies,  Hympavzi (marstacimab-hncq), is a medication indicated for the treatment of patients with Hemophilia A and Hemophilia B.

Hympavzi utilizes a new pathway to help reduce the risk of bleeding episodes in patients with Hemophilia, a disease that impacts the patient’s ability to clot properly. The disease puts patients at risk for prolonged bleeding after an injury or spontaneous bleeding in muscles, joints or organs which can be painful, and in severe cases life-threatening.  

Traditional therapy has involved providing intravenous infusions of clotting factors two to three times per week.  Hympanzi is the first Hemophilia treatment that is available as once-weekly self-administered injection offering significant convenience for patients. 

Due to the nature and cost of the disease (it has been estimated that the lifetime cost for a patient with hemophilia can exceed $20M), Hemophilia is an area of intense research interest. Hympanzi joins traditional therapies and more recently approved one-time gene therapies. Watch for more developments in this exciting area of drug development.

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