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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