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.
Sarepta Therapeutics has announced that the FDA has granted accelerated approval for EXONDYS 51 (eteplirsen) as a once weekly intravenous infusion of 30 milligrams per kilogram for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation in the DMD gene that is amenable to exon 51 skipping. This indication is based on an increase in dystrophin in skeletal muscles observed in some patients treated with EXONDYS 51. A clinical benefit of EXONDYS 51 has not been established. Continued approval for this indication may be contingent upon verification of a clinical benefit in confirmatory trials. The most common adverse reactions compared to a placebo group were vomiting (38%) and balance disorder (38%) with contusion, excoriation, arthralgia, rash, catheter site pain, and upper respiratory tract infection also reported more frequently than placebo (≥ 10%).
“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.”
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