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.
Concizumab-mtci is a subcutaneous therapy, which is developed using a synthetically produced (recombinant) antibody derived from a single cell clone, designed to be equally effective in individuals with hemophilia A and B, irrespective of inhibitor status. It targets an anticoagulant protein known as tissue factor pathway inhibitor (TFPI). It works by blocking and effectively preventing TFPI from performing the anticoagulant function that it naturally carries out in the human body. This type of therapy provides an alternative to traditional prophylaxis with intravenously delivered factor replacement products.
This new approval is based on results from the phase 3 explorer8 trial which showed that concizumab-mtci prophylaxis reduced annualized bleeding rates by 86% in inhibitor-free patients with hemophilia A and 79% in inhibitor-free patients with hemophilia B, when compared with no prophylaxis.
“For people living with hemophilia, it is important to continually monitor and discuss bleed control with their healthcare professional,” said Allison P. Wheeler, MD, scientific director of the Washington Center for Bleeding Disorders at Seattle, WA. “With today’s approval of Alhemo for hemophilia A or B without inhibitors, more people living with these rare blood disorders now have a daily prophylaxis option that may help decrease their bleeding rates.”
Source: Hematology Advisor, August 13, 2025
