On Jul. 31, 2025, Novo Nordisk announced that the US Food and Drug Administration (FDA) approved Alhemo^® (concizumab-mtci) injection as a once-daily prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients 12 years of age and older with hemophilia A or B (HA/HB) without inhibitors, expanding on the December 2024 approval for HA/HB with inhibitors.

Currently, many treatments for HA/HB without inhibitors are administered via intravenous infusions. With this approval, Alhemo^® now offers a subcutaneous injection treatment option for this population.

Alhemo^® is designed to block a protein called tissue factor pathway inhibitor (TFPI), which stops blood from clotting. By inhibiting TFPI, Alhemo^® improves the production of thrombin, which helps to clot the blood and prevent bleeding, when clotting factors VIII and IX are missing or deficient regardless of inhibitor status.

The primary objective of the pivotal phase 3 explorer8 trial was to compare the number of treated spontaneous and traumatic bleeding episodes, as measured by the ABR, in patients aged 12 years and older with HA/HB without inhibitors, receiving Alhemo^® prophylaxis versus no prophylaxis (on demand Factor VIII/Factor IX treatment). 

The study showed a statistically significant reduction in ABR of 79% for HB patients (ABR ratio: 0.21; 95% CI: 0.10–0.45; p<0.0001) and 86% for HA patients (ABR ratio: 0.14; 95% CI: 0.07–0.29; p<0.0001). Additionally, notable reductions in the average and median ABRs further
reinforced the efficacy of Alhemo^® prophylaxis in reducing bleeds across both patient groups