Adam is correct:
The U.S. Food and Drug Administration is actively reviewing the efficacy and safety of Sarepta Therapeutics' (SRPT) Duchenne muscular dystrophy drug eteplirsen even though the drug has not been formally filed for approval yet.
Weird, huh? But also good news for Sarepta. While investors have been obsessing over whether or not FDA will allow an eteplirsen accelerated approval, the agency is already reviewing the drug as if it was filed.
Why? Because FDA understands that a decision to allow Sarepta to file for accelerated approval filing means eteplirsen will be approved. What's going on with eteplirsen now at FDA is not a "file or don't file" decision, it's a mini review.
Read carefully Sarepta's announcement Monday because the evidence of an ongoing eteplirsen review is all there. FDA has asked the company to provide written summaries supporting the use of dystrophin as a surrogate endpoint for approval, as well as a discussion of clinical outcomes from the phase II study.
The FDA doesn't ask for this type of information from companies who haven't yet filed a drug for approval. Instead, these are the types of questions FDA asks of a drug sponsor during the review process.
** Bottom line: The FDA is lining up the data they need to support the approval and the full approval will happen faster than anybody expects **.