FDA expands Hympavzi approval to more hemophilia patients
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Pfizer's Hympavzi (marstacimab-hncq) just received a significant FDA label expansion, now covering hemophilia A and B patients ages 6 and older regardless of inhibitor status, making it the first subcutaneous non-factor treatment for children in this demographic.
With a new label expansion cleared by the U.S. Food and Drug Administration (FDA), Hympavzi (marstacimab-hncq) is now approved for preventing or reducing the frequency of bleeds in all people with hemophilia A or hemophilia B, ages 6 and older, regardless of inhibitor status.
The injectable therapy was previously approved for adults and children with hemophilia A or B, ages 12 and older, who tested negative for inhibitors, a type of neutralizing antibody that can make standard factor replacement therapies less effective. The new label expands the eligible age range and makes the treatment available to people who test positive for inhibitors.
A Milestone for Pediatric Hemophilia Care
The FDA’s move makes Hympavzi the first under-the-skin (subcutaneous) non-factor treatment available to children with hemophilia B, ages 6-11, according to its developer, Pfizer.
Pfizer said it is working on seeking similar label expansions in other countries. In the European Union, a recent label expansion extended the therapy’s use to people with inhibitors, but it remains limited to those aged 12 and older.
“With this expanded approval, we believe Hympavzi can become a transformative option and meet a significant medical need for people living with hemophilia A or B with or without inhibitors ages 6 years and older,” Aamir Malik, chief U.S. commercial officer and executive vice president at Pfizer, said in a company press release. “Particularly for children ages 6 to 11 with hemophilia B who will now, for the first time, have a subcutaneous non-factor treatment available.”
