Pfizer Inc. (PFE) announced that the U.S. FDA has approved HYMPAVZI for additional patient groups: individuals aged 12 and older with hemophilia A or B who develop inhibitors, and children aged 6-11 with or without inhibitors.
HYMPAVZI was first approved on October 11, 2024, for adults and adolescents (greater or equal to 12 years) with hemophilia A or B without inhibitors. The latest approval expands its use to younger pediatric patients and those with inhibitors, making it the first subcutaneous non-factor therapy available for children with hemophilia B.
Hemophilia is a rare genetic bleeding disorder affecting approximately 800,000 people worldwide. Inhibitors—antibodies that neutralize standard factor replacement therapies—pose a significant challenge, occurring in about 20% of hemophilia A and 3% of hemophilia B patients.
HYMPAVZI works by targeting tissue factor pathway inhibitor (TFPI), helping reduce bleeding episodes with a once-weekly subcutaneous injection that does not require routine lab monitoring.
The FDA's expanded approval was supported by data from the Phase 3 BASIS trial, which evaluated HYMPAVZI in patients with hemophilia A or B, both with or without inhibitors. Results showed a 93% reduction in annualized bleeding rates (ABR) compared to on-demand therapy, with efficacy consistent across adult and pediatric populations. Safety findings were in line with prior studies, with no new signals observed.
This HYMPAVZI application was reviewed under FDA Priority Review, and the agency also granted Breakthrough Therapy Designation for routine prophylaxis to prevent bleeding episodes in hemophilia A and B patients.
Aamir Malik, Chief U.S. Commercial Officer and Executive Vice President, Pfizer, stated: "This approval reflects Pfizer's more than 40-year commitment to advancing care and quality of life for people living with hemophilia."
Guy Young, M.D., Director, Hemostasis and Thrombosis Center at Children's Hospital, Los Angeles, added: "For children who have to deal with bleeding episodes from an early age and for people living with hemophilia who develop inhibitors, treatment options have been limited and are often burdensome. A treatment that can reduce bleeding with straightforward, once-weekly administration has the potential to fundamentally change how patients and caregivers approach this disease, offering control with a level of simplicity this community has long needed." PFE has traded between $23.08 and $28.75 over the past year. The stock closed Friday trading (June 5, 2026) at $26.04, up 1.36%. In pre-market trading, the stock is at $25.95, down 0.34%.
For comments and feedback contact: editorial@rttnews.com
Business News
June 05, 2026 16:18 ET A busy week for economic news flow saw a slew of reports being released that reflected the trends in the U.S. labor market. In Europe, economic growth and inflation data gained attention as the European Central Bank and Bank of England head for policy session later in the month. In Asia, the monetary policy session of the Indian central bank was in focus as the country, a major oil importer, reels under the pressures of a weaker rupee and rising inflation.