NAARDEN, Netherlands & WALTHAM, Mass.--(BUSINESS WIRE)-- Prilenia Therapeutics B.V. , a clinical stage biotechnology company focused on the urgent mission to develop novel therapeutics to slow the progression of neurodegenerative diseases and neurodevelopmental disorders, announces the acceptance of its European Marketing Authorisation Application (MAA) for pridopidine (45 mg orally twice daily) for the treatment of adults with Huntington’s disease (HD) by the European Medicines Agency (EMA).
Pridopidine is a highly selective and potent sigma-1 receptor (S1R) agonist with an established safety profile. If approved, pridopidine could be the first treatment for Huntington’s disease with the ability to impact progression. The MAA is based on the totality of evidence for safety and efficacy from pridopidine’s extensive development program.
HD is a genetic, rare, fatal neurodegenerative disease that remains without any approved therapeutic options capable of impacting the progression of the disease. All we have to give patients right now is a small handful of options that may offer some degree of symptom control for HD-related chorea and behavioral complications. Patients deserve better than to slowly and inexorably decline from this devastating disease, and this could be the biggest advance in therapy in years. Pridopidine could, for the first time, provide an option that may slow down decline in several functionally relevant disease domains and thus potentially offer patients and their families an extension to the quality time they have together. Since its safety profile appears favorable and it is taken orally, pridopidine can be easily made available to patients.
Dr. Ralf Reilmann
Board Certified Neurologist and Founding Director of the George-Huntington-Institute in Muenster, Germany .
We have studied pridopidine extensively and have a clear understanding of its efficacy and safety. Pridopidine has delivered consistent efficacy benefits across multiple key measures of HD and has demonstrated a placebo-like safety profile in a large safety database. We have submitted a compelling suite of evidence and pridopidine presents the opportunity for a much-needed paradigm shift in HD therapy.
Dr. Michael R. Hayden
CEO of Prilenia
People with HD are acutely aware of their need for new and better treatment options, especially those that can slow down decline. Complex and rare diseases, like HD, are highly challenging and require forward-thinking, and we applaud the progress regulators are making in trying to support the urgent needs of people with HD. If we can come out of the other end of this process with a new therapeutic approach, offering the potential to slow down disease progression, we will have taken a huge step forward for the whole community.
Astri Arnesen
President of the European Huntington's Association
MAA review typically takes 12-14 months but can take longer. In parallel, Prilenia continues its dialogue with the FDA on a potential path forward for pridopidine in the U.S. The Company will consider regulatory submissions for additional countries and regions following the regulatory review process in Europe.