FDA Approves Alhemo® for Hemophilia A and B Treatment Without Inhibitors

The FDA has approved Alhemo (concizumab-mtci) as a once-daily prophylactic treatment for adults and children 12 years and older with hemophilia A or B without inhibitors. The approval is based on phase 3 trial data, which showed an 86% reduction in annualized bleeding rate in patients with hemophilia A and an 79% reduction in patients with hemophilia B using Alhemo prophylaxis compared to no prophylaxis. This expands on the December 2024 approval for HA/HB with inhibitors.

The European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion, recommending an update to the Alhemo® (concizumab) label to include the treatment of severe haemophilia A and moderate or severe haemophilia B without inhibitors. Pending European Commission (EC) approval, Alhemo® will become available to all adult and paediatric patients 12 years and older living with severe haemophilia A and moderate or severe haemophilia B without inhibitors [1].

The positive opinion is based on results from the explorer8 trial, which demonstrated that Alhemo® reduced spontaneous and traumatic bleeds for patients with haemophilia A and B without inhibitors compared to no prophylaxis. The trial showed an 86% reduction in annualized bleeding rate for haemophilia A and a 79% reduction for haemophilia B [1]. The CHMP's decision follows the recent FDA approval of Alhemo for adults and children 12 years and older with haemophilia A or B without inhibitors, which was based on similar phase 3 trial data [2].

Alhemo®, an anti-tissue factor pathway inhibitor (TFPI) monoclonal antibody, blocks a protein in the body that stops blood from clotting, ensuring the production of thrombin to prevent bleeding. The drug is currently approved in various countries, including the United States, India, Brazil, and Switzerland, for the treatment of adolescents and adults with haemophilia A and B with and without inhibitors [3].

The explorer8 study, a multicentre, open-label, randomised, phase 3a clinical trial, aimed to establish the efficacy and safety profile of Alhemo® in adults and paediatric patients living with congenital severe haemophilia A or moderate or severe haemophilia B without inhibitors [4]. The trial enrolled 148 patients, with 21 receiving no prophylaxis and 42 receiving Alhemo® prophylaxis. The primary endpoint was met, showing significant reductions in treated spontaneous and traumatic bleeding episodes [4].

Following the positive opinion from the CHMP, Novo Nordisk expects the European Commission (EC) to approve the label update within approximately two months. This approval would extend Alhemo®'s convenient once-daily, under-the-skin administration to patients living with haemophilia without inhibitors, providing an efficacious prophylactic choice [1].

References:
[1] https://www.biospace.com/press-releases/european-regulatory-authority-adopts-positive-opinion-for-novo-nordisks-alhemo-concizumab-recommending-label-expansion-to-treat-haemophilia-a-and-b-without-inhibitors
[2] https://www.accessdata.fda.gov/drugsatfda_docs/label/2024/761315s000lbl.pdf
[3] https://www.ema.europa.eu/en/documents/product-information/alhemo-epar-product-information_en.pdf
[4] https://clinicaltrials.gov/study/NCT04082429