Retrophin's Focus Shifts After Phase III PKAN Drug's Failure
Fosmetpantotenate missed all endpoints in a Phase III trial, so Retrophin will shelve the candidate for the rare disease PKAN. Now, the biotech turns focus to Phase III sparsentan in a pair of indications.
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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.