Ketamine Earns FDA Orphan Drug Designation To Treat Amyotrophic Lateral Sclerosis

Release Date: 23-Sep-2021

PharmaTher Holdings Ltd. got permission from orphan drug designation (ODD) for ketamine in the treatment of Amyotrophic Lateral Sclerosis (ALS), also known as Lou Gehrig's disease. Ketamine has the potential to effectively proliferation the life expectancy of those with ALS at any stage and slow the progressive loss of muscle associated with poor outcomes of the disease.


Mechanism of Ketamine is blocking the action of the ionotropic glutamate receptor, the NMDA receptor. Ketamine can lower D-serine concentrations intracellular and also partially activates dopamine receptors. Together it contributes in part to the drug’s neuroprotective effects, which may extend to the motor neurons targeted in ALS.


Currently, the company is working with clinicians in finalizing a phase 2 study protocols to evaluate ketamine for ALS in the U.S. ALS is a progressive neuromuscular disease with a life expectancy of only two to six years after diagnosis. As of now, there is no known treatment for ALS. ALS affects about 50,000 people in the U.S. and Europe, with over 5,000 new cases diagnosed annually.


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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