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FDA has issued a complete response letter to Sarepta Therapeutics for its new drug application seeking approval for a new Duchenne muscular dystrophy drug.
On Aug. 19, 2019, Sarepta Therapeutics announced that it had received a complete response letter (CRL) from FDA regarding its new drug application (NDA) seeking accelerated approval of golodirsen injection for treating Duchenne muscular dystrophy (DMD) in patients with a confirmed mutation amenable to exon 53 skipping.
The CRL 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 was observed in pre-clinical models at golodirsen doses that were ten-fold higher than the dose used in clinical studies. Renal toxicity was not observed in the study on which the application for the drug was based.
“We are very surprised to have received the complete response letter this afternoon. Over the entire course of its review, the [a]gency 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, in a company press release. “We will work with the [d]ivision 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.”
Sarepta will immediately request a meeting with FDA to determine next steps, the company stated.
Source: Sarepta Therapeutics