FDA approves Hympavzi for hemophilia A or B without inhibitors
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The FDA approved marstacimab-hncq for treatment of certain individuals with hemophilia.
The indication applies to routine prophylaxis to prevent or reduce frequency of bleeding episodes among adults or children aged 12 years or older who have hemophilia A without factor FVIII inhibitors. The indication also applies to use of the agent for people with hemophilia B without factor IX inhibitors.
Marstacimab-hncq (Hympavzi, Pfizer) is the first anti-tissue factor pathway inhibitor approved in the United States for treatment of hemophilia A or B, according to a Pfizer press release. It is administered subcutaneously on a once-weekly dosing schedule.
Marstacimab-hncq targets the Kunitz 2 domain of tissue factor pathway inhibitor (TFPI). The natural anticoagulation protein prevents the formation of blood clots and restores hemostasis, according to the release.
The FDA based approval on results of the phase 3 BASIS trial, which included adults and adolescents with hemophilia A or B without inhibitors.
After a 12-month active treatment period, marstacimab-hncq reduced the annualized bleeding rate for treated bleeds by 35% compared with routine prophylaxis and 92% compared with on-demand treatment.
The most common reported adverse events included injection site reactions, headache and pruritus.
“The approval of Hympavzi is a meaningful advancement for people living with hemophilia A or B without inhibitors for bleed prevention, with a generally manageable safety profile and a straightforward once-weekly subcutaneous administration,” Suchitra S. Acharya, MD, director of the hemostasis and thrombosis center at Northwell Health and program head of the bleeding disorders and thrombosis program at Cohen Children’s Medical Center, said in the release. “Hympavzi aims to reduce the current treatment burden by meeting an important need for these patients, including many who have required frequent, time-consuming intravenous treatment infusion regimens.”
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