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Sarepta Therapeutics receives Complete Response Letter for golodirsen

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

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All PolarisDMD sites are now launched

Catabasis announces that all 40 sites, across eight countries, are now fully launched for the Phase 3 PolarisDMD trial of edasalonexent in Duchenne muscular dystrophy!

La Force is happy to share the latest edition of the Catabasis Connection newsletter about the Phase 3 PolarisDMD trial for edasalonexent in Duchenne. The Phase 3 PolarisDMD trial is enrolling boys affected by Duchenne ages 4 to 7, any mutation type, that are not on steroids. This newsletter includes an illustration of the PolarisDMD and GalaxyDMD patient experience.

Clinical trial sites are enrolling rapidly. There is limited space in the United States, Canada and Australia. Locations in the UK, Germany, Ireland, Sweden, and Israel are at capacity. Catabasis anticipates that the final patient screening visits will be completed this September. If you have any questions about the trial > DMDtrials@catabasis.com

Link to the PDF file > https://www.catabasis.com/Catabasis%20Connection%20No.20.pdf

About edasalonexent

Edasalonexent inhibits NF-kB, a protein that plays a fundamental role in skeletal and cardiac muscle disease in Duchenne. By inhibiting NF-kB, edasalonexent has the potential to decrease inflammation and fibrosis, promote muscle regeneration, and slow disease progression. Edasalonexent is being developed as a potential stand-alone therapy and also have the potential to be combined with dystrophin-targeted therapies.

Learn more about edasalonexent

  • Watch this video recorded in November 2016, Dr. Joanne Donovan answers our questions about edasalonexent (CAT-1004)

About Canadian sitesClinical trials in Canada –  PHASE 3 POLARISDMD TRIAL OF EDASALONEXENT IS NOW OPEN FOR ENROLLMENT IN CANADA

About Catabasis

Their mission is to bring hope and life-changing therapies to patients and their families. Their lead program is edasalonexent, an NF-kB inhibitor in development for the treatment of Duchenne muscular dystrophy. Their global Phase 3 PolarisDMD trial is currently enrolling boys affected by Duchenne. For more information on edasalonexent and the Phase 3 PolarisDMD trial, please visit www.catabasis.com or www.twitter.com/catabasispharma.

Stay in touch with Catabasis

Edasalonexent is an investigational drug that is not yet approved in any territory.