The FDA just ap­proved the third Duchenne MD drug. And reg­u­la­tors still don’t know if any of them work

Last year Sarep­ta hit cen­ter stage with the FDA’s con­tro­ver­sial re­ver­sal of its CRL for the com­pa­ny’s sec­ond Duchenne mus­cu­lar dy­s­tro­phy drug — af­ter the biotech was am­bushed by agency in­sid­ers ready to re­ject a sec­ond pitch based on the same dis­ease bio­mark­er used for the first ap­proval for eteplirsen, with­out ac­tu­al da­ta on the ef­fi­ca­cy of the drug.

On Wednes­day the FDA ap­proved the third Duchenne MD drug, based on the same bio­mark­er. And reg­u­la­tors were ready to act yet again de­spite the lack of ef­fi­ca­cy da­ta.

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