Novo Nordisk announced that the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion recommending an expanded label for Alhemo (concizumab), supporting its use in patients with severe haemophilia A and moderate to severe haemophilia B without inhibitors.
The recommendation, if approved by the European Commission, will broaden access to Alhemo beyond its current indication for patients with haemophilia who have developed inhibitors, extending its once-daily subcutaneous prophylactic treatment to a wider population.
Martin Holst Lange, Executive Vice President and Head of Development at Novo Nordisk, highlighted the significance of the potential label expansion: “With its user-friendly, pre-filled, portable pen, Alhemo has the potential to offer more patients a flexible and individualized treatment option that fits their evolving needs.”
The CHMP’s positive opinion is based on results from the Phase 3 explorer8 trial. The study showed that Alhemo® significantly reduced bleeding episodes in patients without inhibitors. Specifically, it led to an 86% reduction in treated spontaneous and traumatic bleeds in patients with haemophilia A, and a 79% reduction in those with haemophilia B, compared to no prophylaxis.
In addition to strong efficacy, Alhemo demonstrated a favourable safety profile. Patient-reported outcomes from the trial also revealed improvements in health-related quality of life and reductions in treatment burden. Survey responses indicated better physical functioning, reduced bodily pain, and a preference for Alhemo over previous treatments—nearly 71% of participants favored it compared to their prior regimen.
Novo Nordisk expects a final decision from the European Commission within approximately two months. If approved, the label expansion will offer a more convenient and effective treatment option to a broader range of people living with haemophilia in the EU.