Should the FDA have approved Eteplirsen?

I should, before continuing, declare my conflict of interest. Dear FDA, I am writing to you about the forthcoming meeting to consider the marketing application for eteplirsen, a morpholino-oligo that induces exon-skipping to generate functional dystrophin protein and therefore, it is hoped, improved outcomes for patients with Duchenne Muscular Dystrophy (DMD). read more

So eteplirsen got “approved” but it’s really a conditional approval. With a lot of conditions. I’m not sure if Sarepta is a company that can be trusted with those conditions but this does give the FDA a lot of leeway in pulling approval if they have to. read more

While the previous delay did give some hope to the idea that there could be approval coming from the FDA, this delay should help to make investors more confident in the approvability of Eteplirsen. The FDA announced that it is delaying an approval decision regarding Eteplirsen, and is asking Sarepta to provide dystrophen data from already obtained biopsies, from the company's ongoing trial. read more