Rare Neurologic Disorder Therapy Granted Orphan Drug Status
Retrophin announced that the Food and Drug Administration (FDA) has granted Orphan Drug designation to its investigational therapy RE-024 (phosphopantothenate replacement therapy), for the treatment of pantothenate kinase-associated neurodegeneration (PKAN).
The company has begun screening healthy volunteers for its recently announced Phase 1 trial with RE-024 (phosphopantothenate replacement therapy). The study has been designed to evaluate the safety and tolerability of single oral doses of RE-024.
Pantothenate kinase-associated neurodegeneration is a rare and lethal autosomal recessive neurodegenerative disorder. Currently there are no approved treatments for this disorder.
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