Prilenia and Ferrer provide update on European Regulatory Process for Pridopidine in Huntington's disease.
We learned on July 25, 2025, that Prilenia’s application to the European Medicines Agency (EMA) for pridopidine was not accepted for marketing authorisation.
Prilenia and Ferrer said,
We are disappointed, but undeterred in our commitment to bring what we believe is an effective therapy to patients and will explore all options collaboratively with regulators. Moreover, we remain fully focused on the ongoing development of pridopidine in HD, ALS and other indications across the world, with plans to undertake a potentially registrational Phase 3 HD study to confirm the previously observed pridopidine results, planned to start in Q2 of next year, and a planned pivotal Phase 3 ALS study expected to start in Jan 2026.
This means pridopidine will not be sold for the treatment of Huntington's disease in Europe.
Official press release:
NAARDEN, The Netherlands and WALTHAM, Mass. / BARCELONA, Spain, 25 July 2025 --- Prilenia Therapeutics B.V. and Ferrer today announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended the refusal of the marketing authorization for pridopidine’s marketing authorization application for Huntington’s disease (HD).
We are disappointed, but undeterred in our commitment to bring what we believe is an effective therapy to patients and will explore all options collaboratively with regulators.
Prilenia and Ferrer are focused on bringing pridopidine to people living with HD and amyotrophic lateral sclerosis (ALS) worldwide as quickly as possible. Near-term plans are in place to initiate a potentially registrational global HD study, to provide confirmation of the previously observed pridopidine results, and a pivotal global Phase 3 ALS study, with recruitment for both expected to start as soon as possible.
About pridopidine
Pridopidine (45 mg twice daily) is a potent and selective, orally administered sigma-1 receptor (S1R) agonist which stimulates key neuroprotective mechanisms often impaired in neurodegenerative diseases such as HD and ALS[i].
Pridopidine’s extensive development program involved approximately 1,600 people, demonstrating clinically meaningful and sustained benefits in disease progression, cognition, motor ability, and quality of life in patients, with a favorable safety and tolerability profile.
In addition to HD, pridopidine is in late-stage clinical development for ALS, with Prilenia and Ferrer planning to initiate a single, pivotal Phase 3 trial in ALS as early as possible, building on the findings in the population with early and rapid progressing disease from the Phase 2 HEALEY ALS Platform Trial.
Pridopidine has Orphan Drug designation in HD and ALS in the US and EU, and FDA Fast Track designation for the treatment of HD[ii].
Nurzigma® (pridopidine) is a registered trademark of Prilenia.
Any failure of a drug is bad news; however, there was uncertainty in the scientific community about the drug. HD Buzz have written an article explaining more about what this means.