by: Parent Project Muscular Dystrophy
PPMD is very disappointed to learn that Sarepta Therapeutics received a Complete Response Letter from the FDA regarding the New Drug Application (NDA) seeking accelerated approval of golodirsen injection for the treatment of Duchenne in patients with a confirmed mutation amenable to exon 53 skipping.
The Complete Response Letter 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. 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.
According to a statement from the company, Sarepta will immediately request a meeting with the FDA to determine next steps. We hope to learn more about these outstanding concerns and to better understand the path forward for our families.
Today has been marked on many of our calendars and like you, we were hopeful that an approval was imminent. One of our toughest challenges in the fight to end Duchenne is time because it is so precious for each of our loved ones affected by this disease. However, we are hopeful that these outstanding concerns can be rectified quickly as Sarepta and the FDA work to bring our community a safe, effective therapy. As always, we extend our deepest gratitude to the families who have participated in golodirsen trials.
Read the Full Release from Sarepta
Sarepta Therapeutics Receives Complete Response Letter from the US Food and Drug Administration for Golodirsen New Drug Application
August 19, 2019 at 5:48 PM EDT
CAMBRIDGE, Mass., Aug. 19, 2019 (GLOBE NEWSWIRE) — Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today 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.
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. 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.
“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,” 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.”
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.
UPDATE:
PPMD invited Sarepta leaders to join a community discussion regarding the New Drug Application for golodirsen on August 21, 2019. Click here to view the recording.
