FDA Approves First Weekly Subcutaneous Therapy for Hemophilia B
Marstacimab-hncq is the first non-factor and once-weekly treatment available for patients with hemophilia B and promises to reduce the burden of care.
The FDA has approved marstacimab-hncq (Hypmavzi) as a prophylactic treatment to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients 12 year of age and older with hemophilia A without factor VIII inhibitors or hemophilia B without factor IX inhibitors, the agency announced in a release.1 The medication is administered weekly using an autoinjector pen and is the first subcutaneous option for patients with hemophilia B.
Platelets forming a blood clot / Катерина Євтехова - stock.adobe.com
Marstacimab-hncq is a novel treatment for hemophilia B. Unlike traditional therapies that replace clotting factors, it works by reducing the amount of anticoagulation proteins called tissue factor inhibitors. This increases the production of thrombin, an enzyme essential for blood clotting, which is expected to reduce or prevent bleeding episodes. Marstacimab-hncq was previously granted Orphan Drug designation by the FDA.1
“Today’s approval of Hypmavzi 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, in the release.1 “This new type of treatment underscores the FDA’s commitment to advance the development of innovative, safe and effective therapies.”
Marstacimab-hncq's approval is based on the results of the phase 3 BASIS trial (NCT03938792), a large-scale clinical study involving 116 male patients with severe hemophilia A or B. Trial results, published in the journal Blood,2 demonstrated the effectiveness of marstacimab-hncq in preventing bleeding episodes.
During the first 6 months of the study, 33 patients received on-demand factor replacement therapy, while 83 patients received treatment with replacement factor prophylactically. After this initial period, all patients received marstacimab-hncq prophylaxis for 12 months. The primary measure of efficacy of marstacimab-hncq was the annualized bleeding rate of treated bleeds.
Patients who received on-demand factor replacement during the first 6 months of the study experienced an estimated annualized bleeding rate of 38 compared to the estimated annualized bleeding rate during treatment with marstacimab-hncq of 3.2. These results suggested that marstacimab-hncq was more effective than on-demand factor replacement in preventing bleeding episodes.
Patients who received prophylactic factor replacement during the first 6 months of the study experienced an estimated annualized bleeding rate of 7.85. After switching to marstacimab-hncq prophylaxis for the following 12 months, the bleeding rate decreased to 5.08. These results showed that marstacimab-hncq demonstrated comparable bleeding rates to prophylactic factor replacement.
Hemophilia A and hemophilia B are genetic bleeding disorders that occur when there is a dysfunction or deficiency of either coagulation factor VIII or IX. In the United States, approximately 33,000 males have hemophilia, with hemophilia A being roughly 3 times more prevalent than hemophilia B.3
Whereas patients with hemophilia B previously required multiple weekly intravenous infusions, marstacimab-hncq is the first non-factor and once-weekly treatment available for the patient population and promises to reduce the burden of care.4
READ MORE: Rare Diseases Resource Center
Don’t get left behind: Sign up today for our free Drug Topics newsletter and get the latest drug information, industry trends, and patient care tips delivered straight to your inbox.
