October 12, 2024 – The U.S. Food and Drug Administration approved Hympavzi (marstacimab-hncq) for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients 12 years of age and older with hemophilia A without factor VIII inhibitors or hemophilia B without factor IX inhibitors (neutralizing antibodies). Product is First Non-Factor and Once-Weekly Treatment for Hemophilia B.
Hympavzi's approval is based on an open-label, multi-center study in 116 adult and pediatric male patients with either severe hemophilia A or severe hemophilia B, both without inhibitors. For the first six months of this study, patients received treatment with replacement factor either on-demand (33 patients) or prophylactically (83 patients). These patients then received Hympavzi prophylaxis for 12 months. The primary measure of efficacy of Hympavzi was the annualized bleeding rates of treated bleeds. In the patients receiving on-demand factor replacement during the first six months of the study, the estimated annualized bleeding rate was 38 compared to the estimated annualized bleeding rate during treatment with Hympavzi of 3.2, showing that Hympavzi was superior to on-demand factor replacement. In the initial six-month period during which patients received prophylactic factor replacement, the estimated annualized bleeding rate was 7.85 and was 5.08 during the subsequent 12 months on Hympavzi prophylaxis, showing that Hympavzi provided similar bleeding rates.
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