Pharma Focus Asia

PharmaTher Holdings Announces FDA Grant of Orphan Drug Designation to KETARX™

Friday, February 03, 2023

PharmaTher 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.

Phase 2 clinical study results to support proposed Phase 3 development of KETARX™ (ketamine) to treat Rett Syndrome for FDA approval via the 505(b)(2) regulatory pathway.

Ketamine is subjected to a Phase 2 clinical trial (NCT03633058) for Rett syndrome. Unpublished results from the study will be evaluated to support a potential Phase 3 clinical study and obtain FDA agreement on a regulatory plan for approval via the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.

The potential of KETARX™ for rare disorders, and the addition of the Rett syndrome programme to the four FDA-approved orphan drug designations strengthens position in leading the advancement of ketamine for unmet medical needs in mental health, neurological and pain disorders.

Orphan drug designation has qualified ketamine for certain benefits and incentives, including seven years of marketing exclusivity if regulatory approval is ultimately received for the designated indication, potential tax credits for certain clinical drug testing costs, eligibility for orphan drug grants, and the waiver of the FDA New Drug Application filing fee of approximately $2.4 million.

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