The Food and Drug Administration (FDA) has granted Orphan Drug designation to pridopidine for the treatment of amyotrophic lateral sclerosis (ALS).

Pridopidine is an investigational oral drug that binds and activates the sigma-1 receptor (S1R), a protein that is highly expressed in the brain. According to the Company, the activation of S1R in the brain and spinal cord results in the clearance of toxic proteins, enhances energy production and neuronal connectivity, and reduces cellular stress and inflammation. The S1R is believed to regulate several cellular mechanisms common to neurodegenerative diseases such as ALS.

The Company is currently evaluating the efficacy and safety of pridopidine for the treatment of ALS in the phase 2/3 HEALEY ALS Platform trial ( Identifier: NCT04297683). Pridopidine has also been shown to have a favorable safety and tolerability profile based on extensive safety data.

“Pridopidine is an exciting compound that offers potential disease modifying treatment for patients with ALS,” said Dr. Jeremy Shefner, MD, PhD, Kemper and Ethel Marley Professor and Chair of Neurology of Barrow Neurological Institute and regimen lead investigator for the pridopidine regimen. “Orphan Drug Designation will speed development and simplify the approval pathway should efficacy be demonstrated in ongoing trials, which will be good news for patients with ALS.”

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Pridopidine is also being evaluated for the treatment of Huntington disease in a phase 3 trial ( Identifier: NCT04556656).


FDA grants Orphan Drug designation for pridopidine for the treatment of amyotrophic lateral sclerosis (ALS). [press release]. Naarden, Netherlands: Prilenia Therapeutics B.V.; July 12, 2021. 

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