Today the U.S. Food and Drug Administration (FDA) approved Exondys 51 (eteplirsen) injection, the first drug approved to treat patients with Duchenne muscular dystrophy. Exondys 51 is specifically indicated for patients who have a confirmed mutation of the dystrophin gene amenable to exon 51 skipping - about 13% of the Duchenne population. Read the FDA's press release.
Edward Kaye, MD, Sarepta's interim chief executive officer and chief medical officer stated, "Today's accelerated approval of Exondys 51 represents a major milestone in the treatment of Duchenne Muscular Dystrophy for patients amenable to skipping exon 51 by targeting the underlying genetic cause of the disease – the lack of the dystrophin protein. 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." Read Sarepta's full press release.
PPMD applauds the FDA for this landmark approval of the first-ever disease-modifying drug to treat Duchenne. Pat Furlong, Founding President and CEO of PPMD stated, ""We applaud Sarepta and the many clinicians and scientists who continue to work tirelessly on this therapy. And we are eternally grateful to all of the young men, and their families, who participated in this trial, and the organizations and advocates who worked collaboratively to make this historic day possible. Their sacrifices have forever changed the Duchenne landscape." Read PPMD's full press release.
The accelerated approval of Exondys 51 is based on the surrogate endpoint of dystrophin increase in skeletal muscle observed in some Exondys 51-treated patients. Under the accelerated approval, Sarepta Therapeutics will be required to conduct a clinical trial to confirm the drug's clinical benefit. The required study will be designed to assess whether Exondys 51 improves motor function of Duchenne patients with a confirmed mutation of the dystrophin gene amenable to exon 51 skipping. The primary endpoint will be the North Star Ambulatory Assessment. We will provide additional details regarding this trial as soon as they become available.