Re: The FDA has changed its mind before on drug approvals & timelines
Your point about the meaning of a CRL is well taken.
The market treated the CRL as though it were a big deal: the day after SRPT's press release (Aug. 20), the stock closed $18 lower and managed to lose another $30 over the next month. When news broke that the FDA approved Vyondys 53, the stock closed over $31 higher.
Here's a recap of the case for anyone interested:
-- Feb. 14, 2019: FDA accepts Vyondys 53 (golodirsen) NDA with priority review. A "regulatory action date" was set for August 19.
-- Aug. 19, 2019: Sarepta receives CRL. At issue: "the risk of infections related to intravenous infusion ports and renal toxicity seen in pre-clinical models of golodirsen."
I have not mentioned another strange FDA case: that involving Sarepta's first approved drug, eteplirsen. The Advisory Committee voted down the drug; a public outcry ensued, and then Janet Woodcock, director of the FDA's Center for Drug Evaluation and Research, ignored the AdCom's recommendation (which isn't common). I followed this case back in 2015. A good recap can be found on Wikipedia under "history".