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European Commission grants orphan drug designation for RE-024
April 2016
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SAN DIEGO—Retrophin Inc.'s RE-024, a novel investigational phosphopantothenate replacement therapy for pantothenate kinase-associated neurodegeneration (PKAN), has been granted orphan drug designation by the European Commission. The compound also received orphan drug designation from the U.S. Food and Drug Administration in 2015, as well as Fast Track status. PKAN is a rare genetic neurological disorder with no approved treatment. It results from a mutation in the PANK2 gene, which encodes a critical protein that phosphorylates vitamin B5 (pantothenate) to phosphopantothenate. This in turn results in decreased levels of Coenzyme A (CoA), which plays a role in many cellular functions. RE-024 is a small molecule in Phase 1 clinical development as a phosphopantothenate replacement therapy for PKAN, and preclinical studies in PANK-deficient animal models and cell lines indicate RE-024 can restore CoA levels.
 
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