Exondys 51 Approval History
- FDA approved: Yes (First approved September 19th, 2016)
- Brand name: Exondys 51
- Generic name: eteplirsen
- 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
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