Before Price Controversy, Emflaza Got Easy US Nod With Old But Good Enough Data
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.
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A year in our long-running series of deep dives into FDA review documents.
US FDA's Search For Meaning: Interpretation Of Regulatory Language Can Inspire Conflict In Drug Reviews
The Pink Sheet's Drug Review Profile series illustrates the challenge of reconciling review realities and broad statutory directives.
Merck's Keytruda may not be only oncologic with indication for cancer patients defined by molecular signature, not tissue of origin, for long: Ignyta's entrectinib and Loxo Oncology's larotrectinib are positioned for breakthrough-designated tissue-agnostic NDA submissions in NTRK fusion-positive cancers in 2018.