FDA Expands Marstacimab Indication in Hemophilia A and B

Phase 3 BASIS and BASIS KIDS data supported the expanded indication across pediatric and inhibitor-pretreated populations.

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The FDA approved marstacimab-hncq (HYMPAVZI) for routine prophylaxis in hemophilia A and B patients aged 6 years and older.
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HYMPAVZI is the first subcutaneous non-factor therapy for pediatric patients aged 6 to 11 years with hemophilia B.
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In the phase 3 BASIS trial, marstacimab reduced the mean annualized bleeding rate by 93% compared to on-demand intravenous treatment.
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Marstacimab targets the Kunitz 2 domain of tissue factor pathway inhibitor to restore balance between bleeding and coagulation.
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Common adverse reactions include injection-site reactions, headache, and pyrexia, with warnings for thromboembolic events and hypersensitivity.

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