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# Retrophin Gains FDA Agreement on Retrophin Phase 3 Trial
- URL: https://www.fdaweb.com/retrophin-gains-fda-agreement-on-retrophin-phase-3-trial/
- Published: 2016-11-11T12:00:00.000Z
- Updated: 2026-09-14T21:46:59.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5137322

Retrophin has reached an agreement with FDA under a Special Protocol Assessment (SPA) for a Phase 3 clinical trial evaluating RE-024, an investigational replacement therapy for treating pantothenate kinase-associated neurodegeneration (PKAN). The SPA provides agency concurrence that the trial’s design can adequately support a future NDA submission. The company says it will begin the the trial before year-end.

The SPA is based on an international, randomized, double-blind, placebo-controlled study evaluating RE-024 in about 82 patients with PKAN aged 6 to 65 years, Retrophin says. The primary endpoint will be the change in score on the Pantothenate Kinase-associated Neurodegeneration Activities of Daily Living (PKAN-ADL) scale, from baseline through 24 weeks of treatment. After completing the 24-week treatment period, all patients will be eligible to receive RE-024 as part of an open-label extension. The PKAN-ADL is a patient-reported outcome scale measuring motor abilities to function in daily living for patients with PKAN.

“Preclinical findings suggest RE-024 has the ability to distribute to the brain and restore CoA levels,” Retrophin says. In a Phase 1 study, RE-024 was found to be safe and well-tolerated in healthy volunteers. In 2015, FDA granted the therapy an orphan drug designation and Fast Track status for treating PKAN.