FDA

Alhemo now approved for hemophilia A/B without inhibitors

August 7, 2025

FDA approved Alhemo (concizumab-mtci) injection as once-daily prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients ≥12 years of age with hemophilia A or B (HA/HB) without inhibitors.

Alhemo had previously been approved for use in HA/HB with inhibitors.

Efficacy

Approval of the new indication was based on results from the explorer8 trial (NCT04082429), which investigated the efficacy and safety of Alhemo for the prophylaxis of bleeding episodes in 118 adult (67 hemophilia A and 51 hemophilia B) and 38 adolescent (23 hemophilia A and 15 hemophilia B) male patients with HA/HB without inhibitors. Primary endpoint was the number of treated spontaneous and traumatic bleeding episodes measured by annualized bleeding rate (ABR).

For patients with hemophilia A, the estimated mean ABR was 2.7 (95% confidence interval [CI], 1.63-4.59) for patients on Alhemo prophylaxis (arm 2) and 19.3 (95% CI, 11.25-33.03) for patients on no prophylaxis (arm 1), corresponding to an 86% reduction in ABR. For patients with hemophilia B, the estimated mean ABR was 3.1 (95% CI, 1.91-5.04) for patients on Alhemo prophylaxis (arm 2) and 14.8 (95% CI, 8.14-26.86) for patients on no prophylaxis (arm 1), corresponding to 79% reduction in ABR.

Safety

The most common adverse reactions reported in the explorer8 trial were injection site reactions (7%) and headache (7%).

Sources:

Alhemo (concizumab-mtci) [package insert]. Novo Nordisk. https://www.novo-pi.com/alhemo.pdf Revised July 2025. Accessed August 6, 2025.

FDA approves Alhemo® as once-daily prophylactic treatment to prevent or reduce the frequency of bleeding episodes for adults and children 12 years of age and older with hemophilia A or B (HA/HB) without inhibitors. [News release]. 2025. https://www.prnewswire.com/news-releases/fda-approves-alhemo-as-once-daily-prophylactic-treatment-to-prevent-or-reduce-the-frequency-of-bleeding-episodes-for-adults-and-children-12-years-of-age-and-older-with-hemophilia-a-or-b-hahb-without-inhibitors-302519177.html