Prilenia: FDA Grants Orphan Drug Designation for pridopidine for the Treatment of Amyotrophic Lateral Sclerosis (ALS)

FDA Grants Orphan Drug Designation for pridopidine for the Treatment of Amyotrophic Lateral Sclerosis (ALS) Prilenia Therapeutics B.V., a clinical stage biotech company focused on developing novel treatments for neurodegenerative and neurodevelopmental disorders, is pleased to announce that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for pridopidine for the treatment of ALS.

Prilenia recently received a positive opinion from the Committee of Orphan Medicinal Products (COMP) in the EU for Orphan Drug Designation for treatment of ALS and is expecting to receive the European Commission decision soon.

Pridopidine acts as a highly selective and potent Sigma-1 receptor (S1R) agonist. Extensive safety data demonstrate pridopidine has a favorable safety and tolerability profile. Pridopidine is currently being assessed in the HEALEY ALS Platform Trial in the U.S., the first for ALS. The pridopidine regimen enrolled its first participant in January 2021 and is on track to generate results in H2 2022. Pridopidine is also being studied for the treatment of Huntington’s Disease (HD) in a global Phase 3 clinical trial that just met the 50% enrollment milestone ahead of schedule.

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