European regulatory authority adopts positive opinion for Novo Nordisk's Alhemo® (concizumab), recommending label expansion to treat haemophilia A and 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.This positive opinion by the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) is based on results from the explorer8 trial, which showed that Alhemo® reduced spontaneous and traumatic bleeds for patients living with haemophilia A and B without inhibitors compared with no prophylaxis1.If approved by the EC, Alhemo®, with its pen-injector device, has the potential to be an efficacious and easy-to-use option for patients with haemophilia A and B without inhibitors1-3. Bagsværd, Denmark, 25 July 2025 – Novo Nordisk today announced that the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion, recommending an update of the Alhemo® (concizumab) label to include the treatment of severe haemophilia A and moderate or severe haemophilia B without inhibitors. “If approved by the European Commission, this label update would extend Alhemo®’s convenient once-daily, under-the-skin administration to patients living with haemophilia without inhibitors, providing an efficacious prophylactic choice,” said Martin Holst Lange, executive vice president and head of Development at Novo Nordisk. “Each person living with haemophilia has individual and evolving needs. With its user-friendly, pre-filled, portable pen, we believe that Alhemo® has the potential to offer even more patients an individualised and flexible treatment.” The positive CHMP opinion is based on the results from the phase 3 explorer8 trial, which met its primary endpoint. The results demonstrated that Alhemo® prophylaxis compared with no prophylaxis treatment led to an 86% reduction and 79% reduction in treated spontaneous and traumatic bleeds for patients living with haemophilia A without inhibitors and haemophilia B without inhibitors1, respectively. In this trial, Alhemo® showed a favourable safety profile in patients with haemophilia A and B without inhibitors1. Patient-Reported Outcome (PRO) data from the explorer8 study further indicated an improvement in health-related quality of life and reduction in treatment burden with Alhemo® treatment compared with no prophylaxis3. Specifically, trends favouring Alhemo® over prophylaxis were observed in the short-form health survey (SF-36v2) and Haemophilia Quality of Life Questionnaire for Adults (Haem-A-QoL) results, in patients with haemophilia A and haemophilia B; these included the change in “bodily pain” and “physical functioning” from baseline to week 24 in the survey, and the Haem-A-QoL “total score” and the “physical health” domain score3. Treatment preference results showed patients were also in favour of Alhemo® over no prophylaxis or previous treatment, with 70.9% of respondents indicating they prefer Alhemo® over their previous haemophilia treatment3. Following the positive opinion from the CHMP, Novo Nordisk expects the European Commission (EC) to approve the label update within approximately two months. 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 studyExplorer8 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 inhibitors1,12. 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 41,12. 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 21,12. Supportive secondary endpoints, such as percent of patients experiencing zero bleeds, are reported as descriptive results only1,12. The trial is still ongoing in the extension phase and is expected to complete in 20281,12. About haemophiliaHaemophilia 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 male14,15. 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 NordiskNovo 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. Contacts for further information Media: Ambre James-Brown +45 3079 9289abmo@novonordisk.comLiz Skrbkova (US)+1 609 917 0632lzsk@novonordisk.comInvestors: Jacob Martin Wiborg Rode+45 3075 5956jrde@novonordisk.comIda Schaap Melvold +45 3077 5649idmg@novonordisk.comSina Meyer +45 3079 6656azey@novonordisk.comMax Ung+45 3077 6414 mxun@novonordisk.com Frederik Taylor Pitter +1 609 613 0568fptr@novonordisk.com _______________________References1. Chowdary P, Angchaisuksiri P, Apte S, et al. Concizumab prophylaxis in people with haemophilia A or haemophilia B without inhibitors (explorer8): a prospective, multicentre, open-label, randomised, phase 3a trial. Lancet Haematol. 2024;11:e891–e904. doi: 10.1016/S2352-3026(24)00307-7.2. Kahr Rasmussen N, Berg B, Christiansen ASL, et al. The Concizumab Pen-Injector is Easy to Use and Preferred by Hemophilia Patients and Caregivers: A Usability Study Assessing Pen-Injector Handling and Preference. Patient Prefer Adherence. 2024;18:1713–1727. doi: 10.2147/PPA.S470091.3. Angchaisuksiri P, von Mackensen S, Apte S, et al. Concizumab prophylaxis in people with hemophilia A or B without inhibitors: patient-reported outcome results from the phase 3 explorer8 study. Res Pract Thromb Haemost. 2025;9:102705. doi: 10.1016/j.rpth.2025.102705.4. Matsushita T, Shapiro A, Abraham A, et al. Phase 3 Trial of Concizumab in Hemophilia with Inhibitors. N Engl J Med. 2023;389:783–794. doi: 10.1056/NEJMoa2216455.5. Alhemo® (concizumab): Summary of Product Characteristics. 2024. Available at: https://www.ema.europa.eu/en/documents/product-information/alhemo-epar-product-information_en.pdf Last accessed: July 2025. 6. Alhemo® (concizumab-mtci): Prescribing Information. 2024. Available at: https://www.accessdata.fda.gov/drugsatfda_docs/label/2024/761315s000lbl.pdf Last accessed: July 2025. 7. CDSCO. List of new drugs (r-DNA origin) approved for import and marketing in India during Jan, 2020 – Apr, 2025 Available at: https://cdsco.gov.in/opencms/opencms/system/modules/CDSCO.WEB/elements/download_file_division.jsp?num_id=MTI3MzI= Last accessed: July 2025. 8. Alhemo® (concizumab): Brazil Product Information. 2025. Available at: https://www.gov.br/anvisa/pt-br/assuntos/medicamentos/novos-medicamentos-e-indicacoes/alhemo-concizumabe-novo-registro Last accessed: July 2025. 9. Alhemo® (concizumab): Switzerland Product Information. 2024. Available at: https://www.swissmedic.ch/swissmedic/en/home/about-us/publications/public-summary-swiss-par/public-summary-swiss-par-alhemo.html Last accessed: July 2025. 10. Alhemo® (concizumab): Japanese Product Information. Available at: https://www.pmda.go.jp/files/000268789.pdf Latest revision date: July 2025. 11. Alhemo® (concizumab): Therapeutic Goods Administration (TGA) Product Information. 2024. Available at: https://www.tga.gov.au/resources/auspar/auspar-alhemo Last accessed: July 2025. 12. ClinicalTrials.gov. NCT04082429. Research Study to Look at How Well the Drug Concizumab Works in Your Body if You Have Haemophilia Without Inhibitors (explorer8). Available at: https://clinicaltrials.gov/study/NCT04082429 Last Accessed: July 2025. 13. Iorio A, Stonebraker JS, Chambost H, et al. Establishing the Prevalence and Prevalence at Birth of Hemophilia in Males: A Meta-analytic Approach Using National Registries. Ann Intern Med. 2019;171:540–546. doi: 10.7326/M19-1208.14. Centers for Disease Control and Prevention. About Hemophilia. Available at: https://www.cdc.gov/hemophilia/about/ Last accessed: July 2025. 15. Statista. Distribution of people with bleeding disorders worldwide in 2020, by gender. Available at: https://www.statista.com/statistics/495675/percentager-of-people-with-bleeding-disorders-in-worldwide-bygender/#:~:text=This%20statistic%20displays%20the%20percentage,with%20hemophilia%20A%20were%20males Last accessed: July 2025. PR250725-Alhemo-CHMP-Positive-Opinion

European regulatory authority adopts positive opinion for Novo Nordisk's Alhemo® (concizumab), recommending label expansion to treat haemophilia A and B without inhibitors

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