Akshat Jain: Marstacimab Gets FDA Expension for Children with Hemophilia B
Akshat Jain, Hematology Pediatric Workforce Task Force at American Society of Hematology, shared a post on LinkedIn:
”Major FDA Milestone for Hemophilia Care: Marstacimab Label Expansion especially Children under 12yrs with Hemophilia B suffering from Inhibitors!
The U.S. FDA has approved an expanded indication for marstacimab, extending its use to:
- Patients with hemophilia A or B aged 12 years and older with inhibitors
- Children aged 6–11 years with hemophilia A or B, with or without inhibitors.
This approval makes HYMPAVZI the first subcutaneous non-factor therapy available for pediatric patients with hemophilia B aged 6–11 years, addressing a longstanding unmet need in the bleeding disorders community.
As a pediatric hematologist, I view this as a particularly important advancement for Hemophilia B families that suffer from inhibitors who have since long being searching for an optimal way to manage bleeding in the setting of high titer inhibitors!
Historically, young patients with inhibitors have faced limited treatment options, significant treatment burden, and challenges maintaining consistent bleed protection.
A once-weekly subcutaneous anti-TFPI therapy offers a meaningful alternative to frequent intravenous infusions while expanding access to effective prophylaxis for a population that has long been underserved.
The approval is supported by data from the Phase 3 BASIS study, which demonstrated a 93% reduction in treated annualized bleeding rates compared with on-demand bypassing therapy in patients with inhibitors.
This milestone reflects the continued evolution of hemophilia care beyond factor replacement, bringing us closer to a future where treatment is more convenient, effective, and patient-centered—especially for children and families navigating the daily challenges of hemophilia.
Hopefully these therapies will be available for the children under 6 yrs suffering from sometimes worse bleeding in early childhood from falls,trauma and sports , very soon.”
Stay updated on all scientific advances with Hemostasis Today.
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