PharmaTher One Step Closer to Commercializing Ketamine To Treat Rett Syndrome

Previous studies have suggested ketamine has the potential to promote synaptic repair in Rett syndrome.

Ketamine pharmaceutical company PharmaTher Holdings Ltd. (OTCQB: PHRRF) (CSE: PHRM) has said that the U.S. Food and Drug Administration has granted orphan drug designation to its proprietary racemic ketamine drug.

The drug has undergone a phase 2 clinical trial for the treatment of Rett syndrome, a rare genetic neurological disorder. The company said that unpublished results from this study will be evaluated to support a potential phase 3 clinical study and obtain FDA agreement on a regulatory plan.

The orphan drug designation means that it would not be profitable to make without government assistance due to the small population of patients affected by such conditions. That assistant translates to certain benefits and incentives, including seven years of marketing exclusivity, potential tax credits for certain clinical drug testing costs, eligibility for certain grants, and the waiver of the FDA’s $2.4 million new drug application filing fee.

“We are committed to unlocking the potential of KETARX for rare disorders, and the addition of the Rett syndrome program to our four FDA-approved orphan drug designations strengthens our position in leading the advancement of ketamine for unmet medical needs in mental health, neurological and pain disorders,” said PharmaTher CEO Fabio Chianelli.

PharmaTher isn’t the only clinical research firm that has opted to take the approach of using psychedelics to treat rare, neurological-based diseases that could receive approval more quickly, as drugs for these diseases often access accelerated regulatory pathways to expedite review processes.

The firm currently holds five orphan drug designations granted by the FDA for racemic ketamine.

There is currently no known cure or FDA-approved drugs for treating Rett syndrome. The genetic disorder occurs almost exclusively in girls and can lead to severe impairments in their ability to speak, walk, eat, and even breathe easily, according to the Rett Syndrome Foundation.

Usually recognized in children between 6 to 18 months, prominent features of Rett syndrome include near constant repetitive hand movements and loss of purposeful hand use — caused by mutations on the X chromosome on a gene mostly found in the brain called MECP2. The syndrome occurs worldwide in 1 of every 10,000 female births and is much rarer in boys.

The company said that previous findings on the treatment of the disorder using ketamine suggest that, in addition to acute rescue of neurological function, ketamine also has the potential to promote synaptic repair by enhancing structural and functional connectivity.

PharmaTher has previously said that it entered into an evaluation and exclusive option agreement with Case Western Reserve University in the development and commercialization of the intellectual property of ketamine in the treatment of the disorder.

Adam Jackson

Adam Jackson covers the cannabis industry for The Green Market Report. He previously covered the Missouri statehouse for The Columbia Missourian and has written for The Missouri Independent. He most recently covered retail, restaurants, and other consumer companies for Bloomberg Business News. You can find him on Twitter @adam_sjackson and email him at

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