EMA Turns Down Nurzigma for Huntington Disease
Huntington disease is a rare, inherited, and fatal neurodegenerative disorder characterized by progressive motor, cognitive, and psychiatric symptoms. It affects approximately 5-10 individuals per 100,000 in the European population, which translates to an estimated 25,000-50,000 cases across the European Union. However, it currently lacks approved therapies to slow its progression. The available treatments only offer limited symptom control for Huntington disease-related complications, including chorea and behavioral issues.
The active substance in Nurzigma, pridopidine, functions as an agonist of the sigma-1 receptor (S1R) within cellular structures, modulating pathways crucial for neuronal health and viability. This action on S1R is thought to ameliorate cellular dysfunctions associated with neuronal injury and Huntington disease, facilitating neurogenesis and attenuating oxidative stress, slowing neurodegenerative processes.
Primary Endpoints Unmet in Trials
Multiple clinical trials — including HART, MermaiHD, and PRIDE-HD — showed that, while pridopidine was generally safe and well tolerated, it failed to meet its primary motor endpoints. Despite this, post-hoc analyses suggested potential benefits in total functional capacity (TFC) in some patients. TFC scores assess how well individuals can perform day-to-day activities, including managing household tasks, finances, work, driving, and cooking.
These findings led to the phase 3 PROOF-HD trial, which focused on functional outcomes rather than motor symptoms. However, the trial did not meet its primary endpoint, which was the change in TFC, in the overall study population. Post-hoc subgroup analyses showed potential benefits, particularly in patients not taking antidopaminergic medications. While these findings offered some insights into potential drug efficacy, the CHMP found them insufficiently robust and lacking in external validity.
Despite setbacks, Prilenia Therapeutics and Ferrer said they are committed to advancing Nurzigma for Huntington disease, with new global studies planned to confirm its effectiveness. The companies have confirmed that the EMA's negative opinion on Nurzigma has no impact on patients currently enrolled in clinical trials or receiving the drug through compassionate use programs. Ongoing access and treatment protocols remain unchanged. However, patients should consult their clinical trial investigators for specific information regarding their continued treatment.
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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 Facebook, Instagram, X, LinkedIn and YouTube. Contacts for further information Media: Ambre James-Brown +45 3079 9289abmo@ Liz Skrbkova (US)+1 609 917 0632lzsk@ Investors: Jacob Martin Wiborg Rode+45 3075 5956jrde@ Ida Schaap Melvold +45 3077 5649idmg@ Sina Meyer +45 3079 6656azey@ Max Ung+45 3077 6414 mxun@ Frederik Taylor Pitter +1 609 613 0568fptr@ _______________________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/ 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: Last accessed: July 2025. 6. Alhemo® (concizumab-mtci): Prescribing Information. 2024. Available at: 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: Last accessed: July 2025. 8. Alhemo® (concizumab): Brazil Product Information. 2025. Available at: Last accessed: July 2025. 9. Alhemo® (concizumab): Switzerland Product Information. 2024. Available at: Last accessed: July 2025. 10. Alhemo® (concizumab): Japanese Product Information. Available at: Latest revision date: July 2025. 11. Alhemo® (concizumab): Therapeutic Goods Administration (TGA) Product Information. 2024. Available at: Last accessed: July 2025. 12. NCT04082429. Research Study to Look at How Well the Drug Concizumab Works in Your Body if You Have Haemophilia Without Inhibitors (explorer8). Available at: 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: Last accessed: July 2025. 15. Statista. Distribution of people with bleeding disorders worldwide in 2020, by gender. Available at: Last accessed: July 2025. Attachment PR250725-Alhemo-CHMP-Positive-OpinionSign in to access your portfolio
