19 August 2019 - Sarepta Therapeutics today announced it had received a complete response letter from the U.S. FDA regarding the new drug application seeking accelerated approval of golodirsen injection for the treatment of Duchenne muscular dystrophy in patients with a confirmed mutation amenable to exon 53 skipping.

The letter generally cites two concerns: the risk of infections related to intravenous infusion ports and renal toxicity seen in pre-clinical models of golodirsen and observed following administration of other antisense oligonucleotides. Renal toxicity with golodirsen was observed in pre-clinical models at doses that were ten-fold higher than the dose used in clinical studies. Renal toxicity was not observed in Study 4053-101, on which the application for golodirsen was based.

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