12 December 2019 -  Vyondys 53 is Sarepta’s second RNA exon-skipping treatment for DMD approved in the U.S.

Sarepta Therapeutics today announced that the U.S. Food and Drug Administration (FDA) has approved VYONDYS 53™ (golodirsen). VYONDYS 53 is an antisense oligonucleotide from Sarepta’s phosphorodiamidate morpholino oligomer (PMO) platform, indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients with a confirmed mutation amenable to exon 53 skipping. 

This indication is based on a statistically significant increase in dystrophin production in skeletal muscle observed in patients treated with VYONDYS 53, which is reasonably likely to predict clinical benefit for those patients who are exon 53 amenable. Consistent with the accelerated approval pathway, the continued approval of VYONDYS 53 may be contingent on confirmation of a clinical benefit in this post-marketing confirmatory trial.

Read Sarepta Therapeutics press release