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The Food and Drug Administration (FDA) has issued a complete response letter (CRL) to Sarepta Therapeutics regarding the New Drug Application (NDA) for golodirsen (SRP-4053), an investigational treatment for patients with Duchenne muscular dystrophy (DMD) with a confirmed mutation amenable to exon 53 skipping.
Golodirsen is a phosphorodiamidate morpholino oligomer (PMO) that works by binding to exon 53 of dystrophin pre-mRNA, resulting in exclusion of this exon during mRNA processing. Exon skipping allows for the production of an internally truncated but functional dystrophin protein. The NDA submission included clinical data from the phase 1/2 4053-101 trial which demonstrated statistically significant results in favor of golodirsen on all biological end points.
The CRL cites 2 concerns: the risk of infections related to intravenous infusion ports and renal toxicity with golodirsen observed in pre-clinical models at doses that were 10-fold higher than the dose used in clinical studies; renal toxicity was not observed in the study included in the NDA submission.
The Company plans to request a meeting with the FDA to discuss next steps. “ Over the entire course of its review, the Agency did not raise any issues suggesting the non-approvability of golodirsen, including the issues that formed the basis of the complete response letter,” said Doug Ingram, president and CEO, Sarepta. “We will work with the Division to address the issues raised in the letter and, to the fullest extent possible, find an expeditious pathway forward for the approval of golodirsen. We know that the patient community is waiting.”
Golodirsen is currently being studied in the Company’s ongoing ESSENCE study, a global, randomized, double-blind, placebo-controlled study evaluating the safety and efficacy of golodirsen and casimersen, an exon-45 skipping agent.
For more information visit Sarepta.com.
