FDA Approves Hympavzi for Certain Adults with Hemophilia A and B

The FDA has approved Hympavzi 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).”

Hemophilia A and hemophilia B are “genetic bleeding disorders caused by a dysfunction or deficiency of coagulation factor VIII (FVIII) or IX (FIX), respectively. Patients with these hemophilias are unable to clot properly and may bleed for a longer time than normal after injury or surgery. They may also have spontaneous bleeding in muscles, joints and organs, which can be life-threatening.”

Hypmavzi 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.”

The drug’s approval is based on a study performed in 116 male patients. 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.”