FDA grants accelerated approval to first drug for Duchenne muscular dystrophy

Approval given to EXONDYS 51 (eteplirsen) as a once weekly intravenous infusion of 30 mg/kg for the treatment of DMD in patients who have a confirmed mutation in the DMD gene that is amenable to exon 51 skipping.

“Today’s accelerated approval of EXONDYS 51 represents a major milestone in the treatment of DMD for patients amenable to skipping exon 51 by targeting the underlying genetic cause of the disease – the lack of the dystrophin protein,” said Edward Kaye, Sarepta’s interim CEO and chief medical officer. “We are grateful to the many patients and investigators who participated in EXONDYS 51’s clinical studies. EXONDYS 51 represents the culmination of many years of work across our entire organization and the Duchenne community to address a critical unmet need by bringing this novel medicine to patients. We will continue to leverage what we have learned from EXONDYS 51 to facilitate future development of potential new treatments targeting additional exons with the goal of one day treating all DMD patients amenable to exon skipping.”