FDA's controversial OK for eteplirsen spurs second thoughts for a rejected rival at BioMarin
Sarepta’s regulatory win for Exondys51 (eteplirsen) left one group of analysts and FDA insiders more than a little flabbergasted.
There was only a minuscule amount of data to look at. And after all, PTC never even got in the doors at the FDA, while the agency had no trouble slapping down BioMarin’s application for drisapersen, which had more data — including its failure in clinical studies and troubling safety signs.
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