- Posted by ISPE Boston
- On August 22, 2019
Sarepta Therapeutics has announced it has received a Complete Response Letter (CRL) from the FDA regarding its New Drug Application (NDA) seeking accelerated approval of golodirsen injection for the treatment of Duchenne muscular dystrophy (DMD). The CRL 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. Sarepta will immediately request a meeting with the FDA to determine next steps.
“We are very surprised to have received the complete response letter…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 chief executive officer, 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.” (Source: Sarepta Therapeutics Website, 19 August, 2019)