Pfizer Inc. announced that the U.S. Food and Drug Administration (FDA) has accepted and granted Priority Review for the company's supplemental Biologics License Application (sBLA) for HYMPAVZI®? (marstacimab) to expand the approved indication to include the treatment of hemophilia A or B patients 6 years and older with inhibitors, and pediatric patients (ages 6 to 11) with hemophilia A or B without inhibitors. In the U.S., HYMPAVZI is currently approved for the treatment of patients 12 years of age and older with hemophilia A without factor VIII (FVIII) inhibitors, or hemophilia B without factor IX (FIX) inhibitors.
The FDA has set a Prescription Drug User Fee Act (PDUFA) action date in the second quarter of 2026. If approved, HYMPAVZI would offer a combination of bleed protection with a straightforward, once-weekly subcutaneous injection administration, requiring minimal preparation and no routine treatment-related lab monitoring for these difficult-to-treat patient populations. The FDA grants Priority Review to medicines that may offer significant advances in treatment or may provide a treatment where no adequate therapy exists.
Priority Review designation by the FDA shortens the standard sBLA review period by four months. The FDA also granted HYMPAVZI Breakthrough Therapy Designation for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in younger pediatric.
