CHMP Recommends Expanding Novo Nordisk’s Alhemo® Access to Broader Haemophilia Population Without Inhibitors

CHMP Recommends Expanding Novo Nordisk’s Alhemo® Access to Broader Haemophilia Population Without Inhibitors

(IN BRIEF) Novo Nordisk has received a positive opinion from the European Medicines Agency’s CHMP recommending label expansion for Alhemo® (concizumab) to include adolescents and adults with haemophilia A and B without inhibitors. Backed by robust data from the explorer8 trial, Alhemo® showed a significant reduction in spontaneous and traumatic bleeding episodes and improved patient-reported outcomes. If approved by the European Commission, Alhemo® could offer a convenient, once-daily subcutaneous prophylactic treatment using a pen-injector, marking a significant step forward for haemophilia management across Europe.

(PRESS RELEASE) BAGSVÆRD, 25-Jul-2025 — /EuropaWire/ — Novo Nordisk has announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion recommending the approval of an updated label for Alhemo® (concizumab). Pending final approval by the European Commission (EC), Alhemo® would become available as a routine prophylactic treatment for adolescents and adults (12 years and older) living with severe haemophilia A or moderate to severe haemophilia B who do not have inhibitors.

The CHMP’s decision is supported by results from the phase 3 explorer8 clinical trial, which demonstrated that Alhemo® significantly reduced both spontaneous and traumatic bleeding episodes compared with no prophylaxis. In patients with haemophilia A and B without inhibitors, Alhemo® lowered treated spontaneous and traumatic bleeds by 86% and 79%, respectively. The once-daily subcutaneous injection, delivered via a convenient pen-injector, offers a flexible and effective solution that could reduce treatment burden for patients.

Martin Holst Lange, Executive Vice President and Head of Development at Novo Nordisk, noted, “If approved, this updated indication will broaden access to Alhemo® as a user-friendly prophylactic option for more people with haemophilia. We believe that the simple administration method and demonstrated efficacy make Alhemo® well suited to support the evolving needs of individuals living with this chronic condition.”

Patient-reported outcomes (PROs) from the explorer8 study revealed improvements in health-related quality of life, with positive trends observed in “physical functioning,” “bodily pain,” and overall scores on the SF-36v2 and Haem-A-QoL instruments. Furthermore, 70.9% of participants expressed a preference for Alhemo® over their previous treatments.

The explorer8 study enrolled 148 participants aged 12 and older with congenital haemophilia A or B without inhibitors. Participants were assigned to receive either no prophylaxis or daily Alhemo® injections, with the primary endpoint focused on the annualised bleeding rate of treated spontaneous and traumatic bleeds. The trial continues in its extension phase and is expected to conclude in 2028.

Alhemo® is a monoclonal antibody that inhibits the tissue factor pathway inhibitor (TFPI), enabling the production of thrombin—a critical component of the blood clotting process. It is currently approved in multiple countries, including the US, Europe, Brazil, India, and Switzerland, for patients with haemophilia A and B with inhibitors, and in Japan and Australia for patients with or without inhibitors.

This positive CHMP recommendation comes as a major step toward enabling broader access to Alhemo® and reinforces Novo Nordisk’s continued investment in developing innovative therapies for people living with haemophilia.

About Alhemo^® (concizumab)
Alhemo^® (concizumab) is an anti-tissue factor pathway inhibitor (TFPI), monoclonal antibody designed to block a protein in the body that stops blood from clotting. By blocking TFPI, Alhemo^® ensures the production of thrombin, which helps to clot the blood and prevent bleeding⁴. Alhemo^® is currently approved in Europe⁵, the United States⁶, India⁷, Brazil⁸ and Switzerland⁹ for the treatment of adolescents and adults (12 years or older) with haemophilia A and B with inhibitors. In Japan¹⁰ and Australia¹¹, Alhemo^® is currently approved for the treatment of adolescents and adults (12 years or older) with haemophilia A and B with and without inhibitors. In all approved countries, it is indicated for routine prophylaxis to prevent or reduce the frequency of bleeding episodes.

About the explorer8 study
Explorer8 is a multicentre, open-label, randomised, phase 3a clinical trial aimed to establish the efficacy and safety profile of Alhemo^® in adults and paediatric patients 12 years of age and older living with congenital severe haemophilia A or moderate or severe haemophilia B without inhibitors^1,12. In explorer8, 148 patients were randomly assigned in a 1:2 ratio to receive no prophylaxis (arm 1, n=21) or Alhemo^® prophylaxis (arm 2, n=42), and 85 were nonrandomly assigned to receive Alhemo^® prophylaxis (arms 3 and 4). The initial loading dose of Alhemo^® was 1 mg per kilogram of body weight, followed by 0.2 mg per kilogram daily, and potentially individualised on the basis of concizumab plasma concentration as measured at Week 4^1,12. The primary analysis was carried out when all patients in arms 1 and 2 completed at least 24 or 32 weeks, respectively, and compared the number of treated spontaneous and traumatic bleeding episodes, measured as annualised bleeding rate, between arms 1 and 2^1,12. Supportive secondary endpoints, such as percent of patients experiencing zero bleeds, are reported as descriptive results only^1,12. The trial is still ongoing in the extension phase and is expected to complete in 2028^1,12.

About haemophilia
Haemophilia is a rare inherited bleeding disorder that impairs the body’s ability to make blood clots, a process needed to stop bleeding. It is estimated to affect approximately 1,125,000 people worldwide¹³. Due to the nature of haemophilia being an x-linked recessive disorder, it often presents differently in males compared with females, with roughly 88% of people diagnosed with haemophilia worldwide being male^14,15. There are different types of haemophilia, which are characterised by the type of clotting factor protein that is defective or missing. Haemophilia A is caused by a missing or defective clotting Factor VIII (FVIII), and haemophilia B is caused by a missing or defective clotting Factor IX (FIX).

About Novo Nordisk
Novo Nordisk is a leading global healthcare company founded in 1923 and headquartered in Denmark. Our purpose is to drive change to defeat serious chronic diseases built upon our heritage in diabetes. We do so by pioneering scientific breakthroughs, expanding access to our medicines, and working to prevent and ultimately cure disease. Novo Nordisk employs about 77,400 people in 80 countries and markets its products in around 170 countries. For more information, visit novonordisk.com, Facebook, Instagram, X, LinkedIn and YouTube.

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SOURCE: Novo Nordisk