FDA expands HYMPAVZI approval

Pfizer>>> announced that the United States — Food and Drug Administration>>> has approved an expanded indication for HYMPAVZI>>> (marstacimab-hncq). This approval allows HYMPAVZI>>> to be used for routine prophylaxis to prevent or reduce bleeding episodes in adults and pediatric patients aged 6 years and older with hemophilia A or B, with or without inhibitors. Previously, HYMPAVZI>>> was approved only for adults and adolescents without inhibitors. The expanded indication is supported by data from the Phase 3 BASIS trial and interim results from the Phase 3 BASIS KIDS trial, which demonstrated significant reductions in annualized bleeding rates. This makes HYMPAVZI>>> the first subcutaneous non-factor therapy available for children with hemophilia B. The application received FDA Priority Review and Breakthrough Therapy Designation, underscoring its potential to address significant unmet medical needs. Aamir Malik>>> of Pfizer>>> and Guy Young>>> of Children s Hospital Los Angeles>>> highlighted the importance of this approval for patients and caregivers.