Novo Nordisk’s Alhemo® Recommended for European Approval as First Once-Daily Subcutaneous Treatment for Haemophilia A and B with Inhibitors

Novo Nordisk’s Alhemo® Recommended for European Approval as First Once-Daily Subcutaneous Treatment for Haemophilia A and B with Inhibitors

(IN BRIEF) Novo Nordisk’s Alhemo® (concizumab) has been recommended for approval by the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP). If approved, Alhemo® will become the first once-daily subcutaneous prophylactic treatment for people aged 12 and older with haemophilia A or B who have developed inhibitors, which reduce the effectiveness of traditional therapies. This new treatment would offer a more convenient alternative to intravenous infusions by allowing patients to use a pre-filled, portable pen for daily administration. The recommendation is based on positive results from the phase 3 explorer7 study, with final approval expected within two months.

(PRESS RELEASE) BAGSVÆRD, 18-Oct-2024 — /EuropaWire/ — Novo Nordisk has announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has issued a positive recommendation for Alhemo® (concizumab). Alhemo® is set to become the first once-daily subcutaneous prophylactic treatment for individuals aged 12 years or older living with haemophilia A or B who have developed inhibitors to standard therapies.

Haemophilia, a rare disorder affecting the blood’s ability to clot, is often treated through intravenous replacement therapy, where missing clotting factors are replaced. However, up to 30% of individuals with severe haemophilia A and 5-10% of those with haemophilia B develop inhibitors, which block the effectiveness of this therapy, leaving patients with limited treatment options. Alhemo® targets these inhibitors by blocking tissue factor pathway inhibitor (TFPI), a protein that impedes blood clotting, allowing clot formation even in the presence of inhibitors.

If granted approval by the European Commission, Alhemo® would offer haemophilia patients a significant advancement in treatment. This once-daily injection is delivered through a convenient, pre-filled pen that can be stored at room temperature for up to four weeks, reducing the treatment burden and providing greater flexibility for patients.

“The CHMP’s positive opinion is a critical milestone for the haemophilia community,” said Stephanie Seremetis, chief medical officer for haemophilia at Novo Nordisk. “Alhemo® offers the potential for easier, everyday prophylaxis to prevent bleeds, alleviating the physical and emotional burden of managing the condition. This is especially significant for patients with haemophilia B and inhibitors, who currently face very limited treatment options.”

The CHMP’s recommendation follows positive data from the phase 3 explorer7 study, which demonstrated the efficacy and safety of Alhemo® in preventing bleeds in people with haemophilia A or B with inhibitors. If approved, Alhemo® will provide patients with a more accessible and manageable alternative to frequent intravenous treatments. Final approval from the European Commission is expected within the next 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 bleeding.5 Alhemo® is currently approved in Australia8 and Switzerland9 for the treatment of adolescents and adults (12 years or older) with haemophilia A or B with inhibitors. In Japan,10 Alhemo® is currently approved for the treatment of adolescents and adults (12 years or older) with haemophilia A or B with and without inhibitors and is indicated for routine prophylaxis to prevent or reduce the frequency of bleeding episodes.

About the explorer7 study
In explorer7, 133 males (aged 12 years or older) were randomised 1:2 to either no prophylaxis (arm one; ≥24 weeks) or concizumab prophylaxis (arm two; ≥32 weeks) or assigned to concizumab prophylaxis (arm three and four).11 The primary analysis compared the number of treated spontaneous and traumatic bleeding episodes, measured as annualised bleeding rate (ABR), between arms one and two. The results showed an 86% reduction in treated spontaneous and traumatic bleeds when on concizumab prophylaxis, with an estimated mean ABR of 1.7 compared to 11.8 with no prophylaxis.12 The overall median ABR of concizumab was zero, compared to 9.8 for no prophylaxis. The safety and tolerability profile of concizumab in this study was within the expected range.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 worldwide.12 Due to the nature of haemophilia being an x-linked recessive disorder, it often presents differently in males compared to females, with roughly 88% of people diagnosed with haemophilia worldwide being male.13,14 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 69,000 people in 80 countries and markets its products in around 170 countries. For more information, visit novonordisk.com, Facebook, Instagram, X, LinkedIn and YouTube.

References

1 Kim JY, You CW. The prevalence and risk factors of inhibitor development of FVIII in previously treated patients with hemophilia A. Blood Res. 2019 Sep;54(3):204-209. doi: 10.5045/br.2019.54.3.204
2 Srivastava A, Santagostino E, Dougall A, et al. WFH Guidelines for the Management of Hemophilia, 3rd edition. Haemophilia. 2020;26 Suppl 6:1-158. doi:10.1111/hae.14046
3 Male C et al. Inhibitor incidence in an unselected cohort of previously untreated patients with severe haemophilia B: a PedNet study. Haematologica. 2021 Jan 1;106(1):123-129. doi: 10.3324/haematol.2019.239160. PMID: 31919092; PMCID: PMC7776246
4 Shapiro AD. Concizumab: a novel anti-TFPI therapeutic for hemophilia. Blood Adv. 2021;5(1):279
5 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 Available at: https://www.nejm.org/doi/full/10.1056/NEJMoa2216455 Accessed: October 2024
6 Frei-Jones M, Cepo K, d’Oiron R, et al. Subcutaneous concizumab prophylaxis in patients with hemophilia A or B with inhibitors: efficacy and safety results by hemophilia subtype from the phase 3 explorer7 trial. Abstract presented at the 64th American Society of Hematology (ASH) annual meeting and exposition 2022.
7 Kahr Rasmussen 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 Preference and Adherence 2024:18 1713–1727
8 Therapeutic Goods Administration (TGA), Product Information. Available at: https://www.tga.gov.au/resources/auspar/auspar-alhemo
9 SwissMedic. Switzerland Product Information. Available at: https://www.swissmedic.ch/swissmedic/en/home/about-us/publications/public-summary-swiss-par/public-summary-swiss-par-alhemo.html
10 Japanese Product Information. Latest revision date: June 2024
11 Jiménez-Yuste V, Angchaisuksiri P, Castaman G, et al. Concizumab prophylaxis in patients with haemophilia A or B with inhibitors: Efficacy and safety results from the 32-week primary analysis of the phase 3 explorer7 trial. Presented at ISTH 2022
12 Iorio A, Stonebraker JS, Chambost H, Makris M, Coffin D, Herr C, Germini F; Data and Demographics Committee of the World Federation of Hemophilia. Establishing the Prevalence and Prevalence at Birth of Hemophilia in Males: A Meta-analytic Approach Using National Registries. Ann Intern Med. 2019 Oct 15;171(8):540-546. doi: 10.7326/M19-1208.
13 Centers for Disease Control and Prevention. What is Hemophilia? Available at: https://www.cdc.gov/hemophilia/about/?CDC_AAref_Val=https://www.cdc.gov/ncbddd/hemophilia/facts.html Accessed: October 2024
14 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 Accessed: October 2024

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Source: Novo Nordisk