FDA Expands Marstacimab Indication in Hemophilia A and B

To report the FDA's approval of an expanded indication for marstacimab-hncq (HYMPAVZI) for routine prophylaxis in hemophilia A and B patients.

In the BASIS trial, marstacimab reduced the mean treated annualized bleeding rate (ABR) by 93% compared to on-demand treatment.
Mean treated ABR was 1.4 for marstacimab versus 19.8 for on-demand treatment (p<0.0001).
In BASIS KIDS, mean treated ABR was 1.8 for patients without inhibitors and 1.4 for those with inhibitors, compared to historical rates of 3.6 and 18.9, respectively.

Marstacimab targets the Kunitz 2 domain of tissue factor pathway inhibitor (TFPI) to restore balance between bleeding and coagulation.

Interim results from BASIS KIDS are descriptive analyses and not inferential comparisons.
Reference rates in BASIS KIDS are historical model-based estimates.

Marstacimab is the first subcutaneous non-factor therapy available for pediatric patients with hemophilia B aged 6 to 11 years.