Sarepta Therapeutics receives Complete Response Letter from the US Food and Drug Administration for golodirsen New Drug Application
Sarepta Therapeutics, Inc announced it had received a Complete Response Letter (CRL) from the U.S. Food and Drug Administration (FDA) regarding the New Drug Application (NDA) seeking accelerated approval of golodirsen injection for the treatment of Duchenne muscular dystrophy (DMD) in patients with a confirmed mutation amenable to exon 53 skipping. – News Release –
What is an FDA Complete Response Letter CRL?
Receiving one of these letters means that the FDA has completed its review of a new drug application and decided not to approve it in its present form.
The U.S. Food and Drug Administration (FDA) sends a complete response letter to communicate it has completed its review of a new or generic drug application, and it decided that it will not approve it for marketing in its present form. Receiving one of these letters from the FDA is never good news, but their long-term impact varies. – The Motley Fool –
The CRL cites two concerns:
- The risk of infections related to intravenous infusion ports
- 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.
Doug Ingram, president and chief executive officer, Sarepta – “We are very surprised to have received the complete response letter this afternoon. 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, 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.”
What is the next step?
Sarepta will immediately request a meeting with the FDA to determine next steps. The ESSENCE study (4045-301), a global, randomized, double-blind, placebo-controlled study assessing the efficacy and safety of golodirsen and casimersen, our exon-45 skipping agent, is ongoing.
More about golodirsen
Like Exondys 51, golodirsen, which Sarepta hopes to sell under the name Vyondys 53, is designed to treat a group of Duchenne patients with a particular type of mutation. Exondys 51 works for about 13% of DMD patients—those whose disease is amenable to exon 51 skipping. If approved, golodirsen would offer treatment to patients with a mutation in exon 53—about 8% of the DMD population.
More interesting links
- Sarepta stumbles on FDA rejection of a new drug to treat Duchenne muscular dystrophy, by Adam Feuerstein
- FDA surprises Sarepta by spurning its 2nd Duchenne drug, by Amirah Al Idrus
- Sarepta Therapeutics completes submission of New Drug Application seeking approval of golodirsen in patients with DMD amenable to skipping exon 53, by La Force DMD
