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news.The US Food and Drug Administration (FDA) has granted fast track designation for Retrophin's new investigational phosphopantothenate replacement therapy, RE-024, to treat pantothenate kinase-associated neurodegeneration (PKAN).
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PKAN is a rare and life-threatening autosomal recessive neurodegenerative disorder.
There are no approved treatments currently available for PKAN, which is believed to affect one to three persons per million across the world.
Currently, the company is enrolling healthy volunteers in its Phase I clinical trial evaluating the safety and tolerability of single oral doses of RE-024.
The FDA’s fast track status accelerates the review process to aid the development of drugs to treat serious health conditions, which do not have proper treatments in place.
The status makes the company eligible for Rolling Review, which allows the submission of completed sections of its new drug application (NDA) for review by the FDA before the company submits a complete application.
Retrophin is a biopharmaceutical firm focused on the development, acquisition and commercialization of drugs for the treatment of serious, catastrophic or rare diseases for which there are currently no viable options for patients.
Approved products of the company include Chenodal, Cholbam, and Thiola.