Nearly two years after their narrow PhIII failure, Orphazyme gets speedy look at the FDA

Orphazyme’s bet that, despite missing both primary endpoints on its pivotal study, they would find welcoming ears at the FDA has paid off, for now.

The Danish biotech announced today that the FDA has accepted the application for their experimental drug arimoclomol in the rare neurodegenerative condition Niemann-Pick disease Type C and given it priority review.