Congratulations ro Pfizer
U.S. FDA Approves Pfizer’s HYMPAVZI™ (marstacimab-hncq) for the Treatment of Adults and Adolescents with Hemophilia A or B Without Inhibitors - Business Wire
HYMPAVZI’s approval is based on Phase 3 study results demonstrating substantial bleed reduction compared to routine prophylaxis and on-demand treatment in eligible patients with hemophilia A or B without inhibitors
In the U.S., HYMPAVZI is the first once-weekly subcutaneous prophylactic treatment for eligible people living with hemophilia B, and the first to be administered via a pre-filled pen or syringe for eligible people living with hemophilia A or B
Results from the Phase 3 BASIS trial (NCT03938792) supported the approval of HYMPAVZI in the U.S. in adults and adolescents with hemophilia A or B without inhibitors. In the study, HYMPAVZI reduced the annualized bleeding rate (ABR) for treated bleeds by 35% and 92% after a 12-month active treatment period compared to routine prophylaxis (RP) and on-demand (OD) treatment, respectively, in patients with hemophilia A or B without inhibitors. The safety profile for HYMPAVZI was consistent with Phase 1/2 results. The most commonly reported adverse reactions (≥3% of patients) in the study were injection site reactions, headache, and pruritus.
"The hemophilia community continually seeks advancements in care that can improve quality of life for our community members," said Phil Gattone, President and CEO, National Bleeding Disorders Foundation. "We greatly appreciate Pfizer's innovative efforts in developing this novel treatment option that addresses some of the ongoing challenges faced by people with hemophilia A and B. The availability of this therapy represents a powerful step forward in advancing care for more individuals and families in the bleeding disorders community.”
The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion for marstacimab for the routine prophylaxis of bleeding episodes in adults and adolescents 12 years and older with severe hemophilia A without FVIII inhibitors, or severe hemophilia B without FIX inhibitors. In addition to HYMPAVZI, Pfizer recently received regulatory approvals for its hemophilia B gene therapy BEQVEZ™ (fidanacogene elaparvovec) in the U.S., EU, and Canada, and announced positive results from a Phase 3 program investigating its hemophilia A gene therapy (giroctocogene fitelparvovec).
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