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FDA Gives Orphan Drug Designation to Ketamine for ALS

Natan Rosenfeld Natan Rosenfeld September 10, 2021
Medically reviewed by Susan Kerrigan, MD and Marianne Madsen

Ketamine is a medication used primarily as an anesthetic in humans and animals. The medication is effective at inducing a state of relaxation and sedation, but its dissociative properties have also made it attractive as a recreational drug. 


However, ketamine has shown potential to treat a wide range of conditions including depression and PTSD. Now, it’s being explored as a treatment for Amyotrophic Lateral Sclerosis (ALS), also known as Lou Gehrig’s disease.


The FDA’s designation


Psychedelics biotech company PharmaTher has been given FDA orphan drug designation (ODD) for their use of ketamine as a potential ALS treatment. The company, which has an “exclusive license agreement” for “the development and commercialization of the intellectual property of ketamine in the treatment of ALS,” produced research data on the molecule which was then evaluated by the FDA, eventually leading to their designation.


When a pharmaceutical is granted orphan drug status, it means the medication has viable potential to treat a rare disease. It also grants a variety of benefits to the drug, including eligibility for grants, tax credits, and marketing exclusivity. PharmaTher’s use of ketamine is on track for a phase 2 clinical trial in the near future.


“Receiving FDA orphan drug designation is a massive validation for ketamine as a potential treatment for ALS, and it allows us to confidently proceed in evaluating ketamine in a phase 2 clinical study in patients suffering from this life-threatening disease,” said Fabio Chianelli, Chief Executive Officer of PharmaTher.


“This designation not only expedites our regulatory, clinical and product development plans, but also validates our belief in the potential of ketamine as a therapeutic solution for neurological disorders.”


Uses of ketamine for ALS


According to researchers at the University of Kansas Medical Center, ketamine is capable of increasing life expectancy in ALS sufferers as well as slowing down muscle degeneration in patients. Furthermore, new preclinical research has shown that ketamine is also able to preserve muscle function in advancing cases of ALS. 


Since today only three medications are FDA-approved for the treatment of ALS (riluzole, edaravone, and Nuedexta), and none of them have the ability to slow disease progression or prolong lifespan, ketamine could be a game-changer for the future of ALS treatment.

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