Concizumab-mtci (Alhemo®) has received approval from the US Food and Drug Administration (FDA) as a once-daily prophylactic treatment for patients aged 12 years and older with hemophilia A or B without inhibitors.

This new approval results in an expanded indication for Concizumab-mtci, which was initially approved by the FDA in December 2024 for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients 12 years of age and older with hemophilia A with factor VIII inhibitors or hemophilia B with factor IX inhibitors.

Explore the latest updates on federal budget reconciliation, healthcare lawsuits, and state-level policy changes impacting bleeding disorders. Learn how NBDF is advocating for access, funding, and patient protections.

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At the turn of the 21st century, there was great excitement and anticipation of gene therapy as a ‘curative’ treatment for hemophilia A and B patients, particularly among those with moderate-to-severe forms of the disease. However, a recent forum piece in the journal Research & Practice in Thrombosis & Haemostasis (RPTH) offers some reasons why the overall commercial uptake of these therapies has been relatively modest, as lingering questions over long-term efficacy (in the case of hemophilia A) and longer-term safety of these first-generation gene therapies.