NAARDEN, Netherlands & WALTHAM, Mass. & BARCELONA, Spain--(BUSINESS WIRE)-- 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 ALSi.
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 HDii.
Nurzigma® (pridopidine) is a registered trademark of Prilenia.
About Huntington’s Disease
Huntington’s disease (HD) is a rare, inherited, autosomal dominant, neurodegenerative disease that results in functional, motor, cognitive and behavioral symptoms. HD is caused by a mutation in the huntingtin geneiii, and each child of a parent with HD has a 50 percent chance of developing the disease.iv
HD affects approximately 4.88 out of 100,000 people around the world with an additional 300,000 people at risk of developing HDv,vi. It is usually diagnosed between the ages of 30 and 50, although HD can occur at any age, including in children and young adults (known as juvenile onset HD or JHD). The disease progresses slowly over 15 to 20 years, with patients slowly losing their ability to work, communicate, manage day-to-day life and take care of themselves. This increasing disability leads to full reliance on a caregiver and, ultimately, death.
The only currently available treatments for HD focus on symptomatic relief and palliative care, with nothing impacting measures of overall progression.
Media Contacts
Prilenia Contact
Communications Team
info@prilenia.com
Ferrer Contact
Alba Soler, Director of Communication
asolerc@ferrer.com