Pfizer Secures Second FDA Approval for Hemophilia Treatment in Six Months

Pfizer's Second Approval:
Pfizer has received its second FDA approval for a hemophilia treatment in six months, with the latest approval being for HYMPAVZI (marstacimab-hncq), a once-weekly subcutaneous prophylactic treatment for adults and adolescents with hemophilia A or B without inhibitors.

Gene Therapy Approval:
Earlier in 2024, Pfizer received FDA approval for BEQVEZ (fidanacogene elaparvovec), an adeno-associated virus (AAV) based gene therapy for the treatment of hemophilia B.

Treatment Details:
HYMPAVZI is an anti-tissue factor pathway inhibitor (anti-TFPI) that targets the Kunitz 2 domain of TFPI to prevent the formation of blood clots and restore hemostasis. It is administered via a pre-filled, auto-injector pen and offers a once-weekly dosing schedule.

Clinical Trial Results:
The FDA approval for HYMPAVZI is based on results from the BASIS study, which showed substantial bleed reduction compared to routine prophylaxis and on-demand treatment in eligible patients with hemophilia A or B without inhibitors.

Pfizer's Commitment:
Pfizer has a long-standing commitment to improving care for people living with hemophilia, with over 40 years of research and development in this area.