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.

For more information visit Retrophin.com.

Want to read more?