Pridopidine for Huntington's Disease Under European Review

Prilenia Therapeutics B.V., a biotechnology company dedicated to developing novel treatments for neurodegenerative diseases and neurodevelopmental disorders, has announced that the European Medicines Agency (EMA) has accepted its Marketing Authorisation Application (MAA) for pridopidine. This drug, taken orally at a dosage of 45 mg twice daily, is intended for the treatment of adults with Huntington’s disease (HD).

Pridopidine is a selective and potent sigma-1 receptor (S1R) agonist known for its safety profile. If the EMA approves it, pridopidine could become the first treatment capable of impacting the progression of Huntington’s disease, a genetic and fatal neurodegenerative disorder that currently lacks approved therapies to slow its progression. Presently, available treatments only offer limited symptom control for HD-related complications, including chorea and behavioral issues.

Dr. Ralf Reilmann, a Board Certified Neurologist and the Founding Director of the George-Huntington-Institute in Muenster, Germany, emphasized the potential breakthrough pridopidine represents. According to Dr. Reilmann, the drug could be a significant advancement for HD patients, offering a chance to slow the decline across several critical areas of the disease and thereby extend the quality time patients can spend with their families. The oral administration and favorable safety profile of pridopidine make it accessible for patients.

Dr. Michael R. Hayden, CEO of Prilenia, highlighted the extensive research conducted on pridopidine, noting that it has shown consistent efficacy across multiple key measures of Huntington’s disease, coupled with a safety profile comparable to a placebo. Dr. Hayden stated that the evidence submitted to the EMA is compelling and that pridopidine could introduce an essential shift in HD therapy.

Astri Arnesen, President of the European Huntington's Association, pointed out the urgent need for improved treatment options for HD patients, particularly those capable of slowing disease progression. Arnesen acknowledged the challenges of addressing complex and rare diseases like HD and praised the regulators’ efforts to meet the urgent needs of the HD community.

The review process for the MAA typically spans 12-14 months, though it can extend further. Concurrently, Prilenia is engaged in discussions with the U.S. Food and Drug Administration (FDA) regarding a potential pathway for pridopidine in the United States. The company also plans to consider regulatory submissions in other regions following the European review.

Pridopidine, taken twice daily at 45 mg, has shown a safety and tolerability profile similar to placebo in clinical studies involving approximately 1,700 patients over up to seven years. The S1R protein, highly expressed in the brain and spinal cord, regulates several processes impaired in Huntington’s disease. Activation by pridopidine stimulates cellular protective pathways crucial for neuronal function and survival, leading to neuroprotective effects.

Prilenia holds Orphan Drug designation for pridopidine for Huntington’s disease and amyotrophic lateral sclerosis (ALS) in both the U.S. and EU. Additionally, the FDA has granted Fast Track designation for pridopidine in treating Huntington’s disease.

Huntington’s disease is a rare, inherited neurodegenerative disorder caused by a mutation in the huntingtin gene. It affects about 100,000 people globally, with an additional 300,000 at risk. The disease typically manifests between ages 30 to 50 and progresses slowly over 15 to 20 years. Currently, no treatments are available to address HD progression.

Founded in 2018, Prilenia is focused on developing therapeutics for neurodegenerative and neurodevelopmental disorders, concentrating initially on Huntington’s disease and ALS. The company, supported by various reputable investors, operates from the Netherlands, Israel, and Massachusetts in the U.S.

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