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Before Price Controversy, Emflaza Got Easy US Nod With Old But Good Enough Data

 
• By Bridget Silverman

Pink Sheet's monthly Drug Review Profile looks at Marathon's (now PTC's) Emflaza for Duchenne muscular dystrophy, and finds that before the steroid's price ignited controversy it received an unremarkable review – unremarkable, that is, for rare and serious illnesses. The US FDA accepted old and flawed trial data because signals of clinical benefit were consistently seen and safety profile was understood.

The US FDA's approval of Marathon Pharmaceuticals LLC's Emflaza (deflazacort) for Duchenne muscular dystrophy quickly set off a controversy about orphan drug pricing and value, but agency review documents show that the decision about the drug's efficacy and safety was anything but controversial.

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