Pridopidine Wins Orphan Drug Designation To Treat ALS

Release Date: 13-Sep-2021

US FDA grants Orphan Drug Designation for pridopidine for the treatment of Amyotrophic Lateral Sclerosis (ALS). Prilenia Therapeutics B.V, a clinical stage biotech company develops pridopidine, a highly selective S1R agonist investigational drug. Pridopidine has a safest and tolerable profile. It 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.


In ALS SOD1G93A motor neurons (MNs), pridopidine exerts neuroprotective effects via activation of the S1R. Specially, pridopidine improves BDNF (brain-derived neurotrophic factor) and GDNF (glial cell line-derived neurotrophic factor) axonal transport, restores synaptic activity and neuro-muscular junction (NMJ) function, and increases neuronal survival. In vivo, pridopidine reduces toxic protein aggregates and ameliorates muscle fiber wasting. 


The FDA's orphan-drug program gives special status to drugs and biologics for diseases and disorders that affect fewer than 200,000 people in the U.S and it also gives several benefits, like tax credits for qualified clinical trials, potential seven years of market exclusivity after approval and exemption from user fees.

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