Despite a strongly critical complete response letter - which the FDA sent in August but did not make public until Tuesday - the drugmaker successfully appealed and won approval for the drug, Vyondys 53, in December, to the surprise of many.
Several FDA approvals - including ones going against AdCom votes - have attracted controversy and a perception of lowered standards at the agency. However, some see a long-standing institutional tension between patient protection and speedy approval, with the latter having a net positive effect.
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In a note to investors, SVB Leerink analyst Joseph Schwartz suggested that the 'questionable' approval of Sarepta's Exondys 51 may have played a role in the FDA's decision to turn down golodirsen.
The decision stood in contrast with the agency's highly controversial approval of Sarepta's previous DMD drug, Exondys 51. The company's shares fell sharply in after-hours trading.
Although the agency goes against about one-fifth of AdCom recommendations, a study published Sunday only found one reliable variable predicting when that would happen.