Ketamine Earns FDA’s Orphan Drug Designation

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by Patricia Inácio, PhD |

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The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to ketamine, PharmaTher’s investigational therapy for amyotrophic lateral sclerosis (ALS).

Orphan drug status is intended to encourage the development of therapies for rare diseases affecting fewer than 200,000 people in the U.S. It provides benefits and incentives, including exemption from FDA application fees, assistance in clinical trial design, and seven years of market exclusivity if eventually approved.

“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,” Fabio Chianelli, CEO of PharmaTher, said in a press release.

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Ketamine is a type of psychedelic or dissociative compound that blocks sensory perception, and is used primarily for starting and maintaining anesthesia. It also is approved for treatment-resistant depression.

Preclinical research, however, reportedly found the compound can safely preserve muscle function and extend survival in animal models of ALS, when given in the early stages of muscle loss. This unpublished and patent-pending research, conducted by researchers at The University of Kansas, was licensed recently by PharmaTher and provided the basis for the FDA’s decision.

In ALS patients, elevated levels of glutamate — a neurotransmitter, or chemical messenger — in the brain and spinal cord cause an overwhelming activation of nerve cells that is toxic and damaging. This occurs partially via glutamate binding to N-methyl-D-aspartate (NMDA) receptors, which overload these neurons with calcium.

Ketamine indirectly blocks these receptors to potentially 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.

Together, these mechanisms may contribute to ketamine being neuroprotective, which could shield the motor neurons that are damaged over the course of ALS.

The company 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. A Phase 2 trial (NCT04912115) in Parkinson’s disease also is planned to begin shortly.

“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,” said Chianelli.