Novo Nordisk’s Alhemo (concizumab-mtci) has been approved by the US Food and Drug Administration (FDA) to prevent or reduce bleeding episodes in a broader population of haemophilia A and B patients.
The therapy has now been authorised for use as a once-daily prophylaxis in adult and paediatric patients aged 12 years and older with haemophilia A or B without inhibitors, building on its existing indication for haemophilia A or B patients with inhibitors.
Affecting approximately 800,000 people globally, haemophilia is a rare inherited bleeding disorder that impairs the body’s ability to make blood clots.
The condition is usually treated with regular intravenous infusions of clotting factor replacement products. However, some patients can develop inhibitors as an immune response to the clotting factors in these therapies, limiting treatment options.
Administered as a subcutaneous injection, Novo’s Alhemo is a tissue factor pathway inhibitor antagonist that is designed to help the blood to clot and prevent bleeding, regardless of inhibitor status.
Anna Windle, senior vice president, clinical development, medical and regulatory affairs, Novo Nordisk, said: “The FDA approval of an expanded indication for Alhemo marks a meaningful step forward for people with haemophilia A or B without inhibitors who are looking for a new prophylaxis treatment option.”
The regulator’s latest decision on the therapy was based on results from the late-stage explorer8 trial, which demonstrated a statistically significant reduction in annualised bleeding rate (ABR) of 79% for haemophilia B patients without inhibitors and 86% for haemophilia A patients without inhibitors, compared to no prophylaxis.
There were also notable reductions in the average and median ABRs, which Novo said “further reinforced the efficacy of Alhemo prophylaxis in reducing bleeds across both patient groups”.
Also welcoming the decision, Allison Wheeler, scientific director, Washington Center for Bleeding Disorders, said: “For people living with haemophilia, it is important to continually monitor and discuss bleed control with their healthcare professional.
“With [this] approval of Alhemo for haemophilia 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.”