Sarepta Therapeutics was “surprised” to receive a complete response letter from the FDA for its new drug application for Vydonos 53 (golodirsen) for treatment of Duchenne muscular dystrophy.
The agency’s decision date for the NDA was Aug. 19. But instead of an approval, it issued a CRL citing the risk of infections from intravenous infusion ports and renal toxicity seen in pre-clinical models.
“Over the entire course of its review, the agency did not raise any issues suggesting non-approvability of golodirsen,” said Sarepta CEO Doug Ingram. He said the company will immediately reach out to the agency to discuss next steps.