Marstacimab-hncq was approved by the U.S. Food and Drug Administration (FDA) as a prophylactic treatment for bleeding episodes in patients 12 and older with hemophilia A without factor VIII inhibitors or hemophilia B without factor IX inhibitors.
The treatment is an anti-tissue factor pathway inhibitor that works by reducing the amount of anticoagulation protein and increasing the amount of thrombin. It is a subcutaneous, once-weekly treatment.
Approval was granted based on the results of an open-label, multicenter phase III trial which demonstrated that 12 months of treatment with marstacimab-hncq reduced annualized bleeding rates by 35% and 92% compared to routine prophylaxis and on-demand treatment, respectively.
“Today’s approval of [marstacimab-hncq] 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 Ann Farrell, MD, director of the Division of Non-Malignant Hematology in the FDA’s Center for Drug Evaluation and Research.
