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.

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