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FDA refuses investigator trials of PKAN drug; Retrophin hit

This article was originally published in Scrip

Executive Summary

Shares of Retrophin plummeted almost 20% on 14 April after the company revealed the FDA is refusing to clear investigational new drug applications (INDs) for investigator-sponsored trials of RE-024, an phosphopantothenate replacement therapy, for pantothenate kinase-associated neurodegeneration (PKAN), a rare and life-threatening neurological disorder.

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