Sarepta Theraputics has announced the reception of a Complete Response Letter (CRL) from the US Food and Drug Administration, (FDA) regarding accelerated approval of golodirsen injection.
The governing body rejected the New Drug Application (NDA) for the Duchenne muscular dystrophy (DMD) drug, citing two major 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 with golodirsen was observed in pre-clinical models at doses that were ten-fold higher than the dose used in clinical studies. Renal toxicity was not observed in Study 4053-101, on which the application for golodirsen was based.
The company say they are “surprised” to have received the letter, 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.”
Doug Ingram, president and chief executive officer explained that the company “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.”
Sarepta has announced that it will immediately request a meeting with the FDA to determine next steps.