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# Sarepta Golodirsen Complete Response Letter
- URL: https://www.fdaweb.com/sarepta-golodirsen-complete-response-letter/
- Published: 2019-08-20T12:00:00.000Z
- Updated: 2026-09-15T01:44:49.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5144809

FDA has issued a complete response letter (CRL) to Sarepta for its NDA seeking accelerated approval of golodirsen injection for treating Duchenne muscular dystrophy in patients with a confirmed mutation amenable to exon 53 skipping. The company [says](https://investorrelations.sarepta.com/static-files/94fb2658-a7ce-433d-88dc-a690a3075691?ref=fdaweb.com) the CRL generally raises 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.

The company expressed surprise at receiving the CRL, stating that “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.” It said it would immediately request a meeting with the agency to determine next steps.