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Exondys 51 Approval History

  • FDA approved: Yes (First approved September 19th, 2016)
  • Brand name: Exondys 51
  • Generic name: eteplirsen
  • Dosage form: Injection
  • Company: Sarepta Therapeutics
  • Treatment for: Duchenne Muscular Dystrophy

Exondys 51 (eteplirsen) is an antisense oligonucleotide indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping.

The accelerated approval of Exondys 51 was based on the surrogate endpoint of increase in dystrophin in skeletal muscle observed in some patients, which the FDA concluded was reasonably likely to predict a clinical benefit, but is requiring further studies to confirm.

Exondys 51 is an intravenous infusion administered over 35 to 60 minutes once weekly. The most common side effects include balance disorder and vomiting.

Development History and FDA Approval Process for Exondys 51

DateArticle
Sep 19, 2016Approval FDA Approves Exondys 51 (eteplirsen) for Duchenne Muscular Dystrophy
Jun  6, 2016Sarepta Therapeutics Announces FDA Request For Dystrophin Data Prior To Making A Decision on Eteplirsen NDA
May 25, 2016Sarepta Therapeutics Announces FDA Will Not Complete the Review of the Eteplirsen NDA By The PDUFA Date
Apr 25, 2016Sarepta Issues Statement on Advisory Committee Outcome for Use of Eteplirsen in the Treatment of Duchenne Muscular Dystrophy
Feb  8, 2016Sarepta Therapeutics Receives Notification of PDUFA Extension for Eteplirsen
Aug 25, 2015Sarepta Therapeutics Announces FDA Has Filed Eteplirsen NDA for the Potential Treatment of Duchenne Muscular Dystrophy for Patients Amenable to Exon 51 Skipping
Jun 29, 2015Sarepta Therapeutics Completes NDA Submission to FDA for Eteplirsen
Oct 27, 2014Sarepta Therapeutics Announces Regulatory Update on Eteplirsen
Apr 21, 2014Sarepta Therapeutics Announces Plans to Submit NDA to FDA for Eteplirsen for Duchenne Muscular Dystrophy
Nov 12, 2013Sarepta Therapeutics Announces FDA Considers NDA Filing for Eteplirsen Premature in Light of Recent Competitive Drug Failure and Recent DMD Natural History Data
Jul 25, 2013Sarepta Therapeutics Announces Plans to Submit New Drug Application to FDA for Eteplirsen for the Treatment of Duchenne Muscular Dystrophy in First Half of 2014

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