Prilenia receives positive EMA opinion on Orphan Drug Designation for pridopidine in ALS

Prilenia Therapeutics B.V., a clinical stage biotech company focused on developing novel treatments for neurodegenerative and neurodevelopmental disorders, received a positive opinion on European Orphan Drug Designation for pridopidine in Amyotrophic lateral sclerosis (ALS). The orphan designation is expected to be granted within 30 days, following the positive opinion issued by the EMA Committee for Orphan Medicinal Products (COMP).

ALS (also known as motor neuron disease and Lou Gehrig disease) is a life-threatening and chronically debilitating disease due to progressive degeneration of motor neurons, ultimately leading to paralysis and respiratory failure.

Pridopidine is a potent and selective Sigma-1 receptor (S1R) agonist, currently being assessed in the HEALEY ALS Platform trial in the US; a multi-center, multi-regimen clinical study evaluating the safety and efficacy of investigational products for the treatment of ALS. The ongoing trial enrolled its first participant for the pridopidine regimen in January 2021 and is on track to generate results in Q3 2022.

The S1R is a promising therapeutic target for the treatment of ALS, based on human genetic data as well as compelling preclinical evidence. Pridopidine demonstrates beneficial effects in key processes relevant to ALS and shows neuroprotective properties in numerous ALS preclinical models, via activation of the S1R.

The COMP granted Orphan Drug Designation for pridopidine in ALS based on supportive preclinical data indicating potential preservation of motor function, which is considered by the committee as an unmet need and a clinically relevant advantage over available treatment.

Under orphan designation in the European Union (EU), Prilenia would benefit from this designation with protocol assistance, reduced regulatory fees and market exclusivity. Orphan Drug Designation in Europe is granted for a serious or life-threatening disease affecting not more than five in 10,000 people.

Dr. Michael R. Hayden, CEO and Founder of Prilenia, commented: “This positive opinion from the EMA further validates the potential for pridopidine to impact the devastating course seen in patients with ALS. We are encouraged by pridopidine’s diverse effects favorably impacting neurodegeneration in ALS models and look forward to the trial readout later next year”.

Prilenia holds Orphan Drug Designation for pridopidine in Huntington’s Disease (HD) in both the US and EU. In addition to the ALS trial, Pridopidine is being studied in a global phase 3 trial for HD (PROOF-HD).