Oct. 14, 2024 – The FDA has approved a brand new preventative treatment for individuals with certain varieties of hemophilia This can reduce bleeding episodes, sometimes even to simply just a few per 12 months.
Hemophilia is a rare blood clotting disorder that could cause prolonged bleeding after an injury or surgery, in addition to spontaneous bleeding into muscles, joints, or organs. The recent treatment, called Hympavzi, is geared toward people aged 12 and over with hemophilia A or hemophilia B. These are the 2 commonest varieties of the condition and are different due to specific proteins involved in blood clotting.
The medicine is injected once per week using a pre-filled pen and offers a substitute for current prevention options that involve infusions several times per week. Its generic name is marstacimab.
“Today’s approval of Hympavzi offers patients with hemophilia a new treatment option that is the first of its kind to target a protein in the blood clotting process,” says Ann Farrell, MD, director of the Division of Nonmalignant Hematology on the FDA Center for Drug Evaluation and Research said in a press release. “This new type of treatment underscores the FDA’s commitment to advancing the development of innovative, safe and effective therapies.”
Instead of supporting normal blood clotting by increasing its clotting ability through infusions, Hympavzi reduces the quantity of a naturally occurring protein that thins the blood. The body then has more enzyme called thrombin, which is significant for blood clotting, which ultimately results in less bleeding.
The approval relies on results of a clinical trial involving 116 men and boys, all of whom had hemophilia A or hemophilia B with severe symptoms, meaning they bled after an injury and had regular spontaneous bleeding without an obvious cause. Thirty-three of them used standard treatments as needed for six months once they were bleeding, and the opposite 83 used standard preventive treatments. All participants within the study then took Hympavzi preventatively for the following 12 months.
Researchers compared the estimated annual variety of bleeding episodes treated through the standard six-month treatment period with the annual rate when people within the study took Hympavzi. Standard as-needed treatment equates to a median of 38 episodes per 12 months, in comparison with 3.2 per 12 months for Hympavzi, the FDA reported in its approval notice.
Standard preventive treatments averaged 7.85 episodes per 12 months, in comparison with 5.08 per 12 months for Hympavzi. The FDA described this rate for Hympavzi as “similar” to the speed for the usual preventive treatments utilized in the study.
In one Press releaseDrugmaker Pfizer said Hympavzi reduced the annual treated bleeding rate by 35% within the group that originally received standard preventive treatments and by 92% within the group that originally received rescue treatments.
Side effects from using Hympavzi may include injection site reactions, headaches, and itching. The FDA also noted that individuals using Hympavzi should consider warnings and precautions regarding circulating blood clots (also called thromboembolic events), hypersensitivity, and possible toxic effects on a developing embryo or fetus.