Strategic Patient Advocacy vs. Evidence Package: The “Rare” Challenge for Orphan Drug Manufacturers

On September 19, 2016, the USA FDA granted accelerated approval to $SRPT (SAREPTA THERAPEUTICS) lead product, EXONDYS 51™(eteplirsen), the first targeted treatment for (DMD), a devastating rare disease. What makes the case unusual is that in approving EXONDYS 51™, the FDA went against the recommendation of its Advisory Committee, a…

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