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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.

"The benefits of deflazacort plainly outweigh its risks," Office of Drug Evaluation I Deputy Director Robert Temple concluded in his Emflaza review memo. "Beneficial effects have been shown on the...

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Potency Assay Issues Delayed US FDA Approval Of Mesoblast’s Ryoncil For Years

 
• By Sue Sutter

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Mesoblast’s Ryoncil: US FDA Changed Its Mind On Need For A Randomized Trial

 
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US FDA Meeting Drought Ends … With A Vengeance

 
• By Michael McCaughan

The first 100 days of the Trump Administration were notable for an extraordinary decline in the number of public meetings hosted by the US Food and Drug Administration. But the schedule changed quickly.

EU CHMP Opinions And MAA Updates

 
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HHS Rescinds COVID-19 Vaccine Advice, Usurping US CDC Role

 
• By Sue Sutter

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