Novel therapy targets blood-clotting proteins to prevent bleeding episodes.
The U.S. Food and Drug Administration (FDA) has approved Hympavzi (marstacimab) as a novel treatment for hemophilia. The drug is designed to prevent or reduce bleeding episodes in patients aged 12 and older with hemophilia A or B who do not have inhibitors to factor VIII or IX. Unlike traditional treatments, which replace missing clotting factors, Hympavzi works by targeting a protein involved in blood clotting, boosting thrombin levels to aid in clot formation.
“Hympavzi provides patients with hemophilia a new treatment option that is the first of its kind to work by targeting a protein in the blood clotting process,” said Director Ann Farrell of the Division of Non-Malignant Hematology in the FDA’s Center for Drug Evaluation and Research.
The approval was based on a clinical trial in which patients first received conventional treatment with clotting factors before transitioning to Hympavzi. The results showed that those treated with Hympavzi experienced significantly lower annualized bleeding rates compared to those using on-demand factor replacement.
As the Lord Leads, Pray with Us…
- For Director Farrell as she oversees the Division of Non-Malignant Hematology.
- For Secretary Becerra as he heads the Department of Health and Human Services.
- For Commissioner Califf and FDA officials as they assess the drugs and treatments for approval.
Sources: Food and Drug Administration
