Orphan Drug Designation Grants Ketamine To Treat ALS

Release Date: 22-Sep-2021

US FDA has granted orphan drug designation to Ketamine for the treatment of amyotrophic lateral sclerosis (ALS). Ketamine is a type of psychedelic or dissociative compound that blocks sensory perception, and is used mainly for starting and maintaining anesthesia. Ketamine can safely preserve muscle function and extend survival in animal models of ALS, when given in the early stages of muscle loss. Also, it is approved for treatment-resistant depression.


Ketamine indirectly blocks these receptors to possibly inhibit glutamate toxicity. It also lowers the levels of cellular D-serine and partially activates dopamine receptors. Elevated D-serine and low dopamine levels are both associated with ALS.

Collectively, these processes may add to ketamine being neuroprotective, which could shield the motor neurons that are damaged over the course of ALS.


PharmaTher’s is planning to initiate a Phase 2 clinical trial to investigate ketamine as a potential treatment for ALS patients. It is in the stage of protocol discussions with clinicians. Shortly, a Phase 2 trial is planned in Parkinson’s disease.


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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