PharmaTher Holdings Announces FDA Grant of Orphan Drug Designation to KETARX (Ketamine) for the Treatment of Rett Syndrome

PharmaTher Holdings Ltd. announced that the U.S. Food and Drug Administration (“FDA”) has granted orphan drug designation to KETARX (racemic ketamine) for the treatment of Rett Syndrome, a rare genetic neurological disorder. Ketamine has been subject to a Phase 2 clinical trial (NCT03633058) for Rett syndrome. Unpublished results from this study will be evaluated to support a potential Phase 3 clinical study and obtain FDA agreement on a regulatory plan for approval via the 505(b)(2) regulatory pathway.

PharmaTher currently holds five orphan drug designations granted by the FDA for KETARX™ (racemic ketamine), which include:

1. Treatment of Rett Syndrome;
2. Prevention of Ischemia-reperfusion injury from organ transplantation;
3. Treatment of Status Epilepticus;
4. Treatment of Amyotrophic Lateral Sclerosis; and
5. Treatment of Complex Regional Pain Syndrome.

“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 Fabio Chianelli, CEO of PharmaTher.