Oral Ketamine Gets Orphan Drug Status for Rett Syndrome

The Food and Drug Administration (FDA) has granted Orphan Drug designation to Ketarx^™ (racemic ketamine) for the treatment of Rett syndrome.

Rett syndrome is a rare genetic neurodevelopmental disorder that occurs primarily in girls. The disorder is characterized by near constant repetitive hand movements and loss of purposeful hand use in addition to loss of coordination and communication abilities. 

There are currently no FDA-approved treatments for Rett syndrome. Data from a phase 2 trial (ClinicalTrials.gov Identifier: NCT03633058) assessing the safety and efficacy of oral ketamine in patients with Rett syndrome is being evaluated to support a potential phase 3 trial.  

“We are committed to unlocking the potential of Ketarx for rare disorders, and the addition of the Rett syndrome program to our 4 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 Fabio Chianelli, CEO of PharmaTher.

Ketarx is also being investigated for prevention of ischemia-reperfusion injury from organ transplantation and for treating status epilepticus, amyotrophic lateral sclerosis, and complex regional pain syndrome.

This article originally appeared on MPR