Orphan drug status sought for ketamine as a potential treatment for ALS


PharmaTher has filed an application with the United States Food and Drug Administration (FDA) requesting that ketamine be designated as an orphan drug as a potential treatment for amyotrophic lateral sclerosis (ALS).

Orphan drug status is granted by the FDA to encourage the development of therapies for conditions that affect fewer than 200,000 people in the United States. receive regulatory approval.

PharmaTher recently entered into an exclusive license agreement with the University of Kansas to develop and eventually commercialize ketamine, approved as a dissociative agent [psychedelic] anesthetic and for treatment-resistant depression, also to treat ALS. He plans to potentially open a Phase 2 clinical trial of ketamine as a treatment for ALS later this year.

“Ketamine has enormous potential not only for mental illnesses and painful disorders, but also for neurodegenerative diseases such as ALS,” Fabio Chianelli, CEO of PharmaTher, said in a press release.

“Our orphan drug application to the FDA for ketamine to treat this potentially fatal disease complements our patent portfolio which seeks to protect the proposed method of use and assay claims of ketamine for ALS,” added Chianelli. “[O]Our intention to seek orphan drug designation is to take advantage of the regulatory and accelerated clinical development incentives offered by the FDA for drugs reused in the treatment of rare diseases.

While ketamine is primarily used as an anesthetic, work from researchers at the University of Kansas suggests that it may also be an effective treatment for ALS.

Their preclinical studies showed that ketamine preserves muscle function, potentially increasing life expectancy when given in the early stages of muscle decline, the company reported.

In patients with ALS, elevated levels of glutamate, a chemical messenger, in the brain and spinal cord produce an overwhelming activation of nerve cells which is toxic. The activation of these nerve cells by glutamate takes place in part by the N-methyl-D-aspartate (NMDA) receptors. Ketamine indirectly blocks these receptors to potentially inhibit the toxicity of glutamate.

Patients with ALS also have high levels of D-serine and low levels of dopamine. Ketamine can decrease D-serine levels and partially activate dopamine receptors.

Overall, these mechanisms contribute to the neuroprotective effects of ketamine, which may enhance the function of damaged motor neurons in ALS, PharmaTher said.

To date, three drugs have been approved by the FDA to treat ALS, and each has marginal effects in slowing the course of the disease and helping patients survive, the company noted. Therefore, there is an urgent need to develop more effective therapies for ALS.

PharmaTher also plans to study ketamine as a treatment for Parkinson’s disease if it is cleared by the FDA for clinical testing.

“We are working with our clinical and regulatory advisors to prepare our clinical development plan and study protocol… to conduct a potential Phase 2 clinical study in patients with ALS this year,” said Chianelli.


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