The FDA has granted Fast Track designation for RE-024, the Retrophin’s novel investigational phosphopantothenate replacement therapy, for the treatment of pantothenate kinase-associated neurodegeneration (PKAN).
PKAN is a rare and lethal autosomal recessive neurodegenerative disorder believed to affect approximately one to three persons per million worldwide, with no approved treatment currently available. Retrophin currently is enrolling healthy volunteers in its phase I clinical trial evaluating the safety and tolerability of single oral doses of RE-024.
Fast Track designation is intended to aid the development of drugs to treat serious conditions with unmet medical needs, and expedite the review process with a goal of reaching patients earlier. A drug that receives Fast Track designation may be eligible for more frequent communication with the FDA to discuss development plans and clinical trial design, as well as Accelerated Approval and Priority Review. The developing company may also be eligible for Rolling Review, which enables the submission of completed sections of its New Drug Application (NDA) for review by the FDA before the company submits a complete application.