FDA approves new treatment for hemophilia A or B

Oct. 14, 2024
Product is first non-factor and once-weekly treatment for hemophilia B.

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).

Hympavzi is a new type of drug that, rather than replacing a clotting factor, works by reducing the amount, and therefore, the activity of, the naturally occurring anticoagulation protein called tissue factor pathway inhibitor. This increases the amount of thrombin, an enzyme that is critical in blood clotting, that is generated. This is expected to reduce or prevent the frequency of bleeding episodes.  

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.

Hympavzi comes with warnings and precautions about circulating blood clots (thromboembolic events), hypersensitivity and embryofetal toxicity. 

The most common side effects of Hympavzi are injection site reactions, headache and itching (pruritis). 

The FDA granted Hympavzi Orphan Drug designation for this application.  

The FDA granted the approval of Hympavzi to Pfizer Inc.

FDA release

matawee matipano/iStock/Getty Images Plus/Getty Images
gettyimages1176254834
ID 335772838 © Yulia Ryabokon | Dreamstime.com
dreamstime_xxl_335772838
ID 3961118 © Sebastian Czapnik | Dreamstime.com
dreamstime_xxl_3961118
ID 12187971 © Valentyn75 | Dreamstime.com
dreamstime_xxl_12187971