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