Industry Partner News: Sarepta Therapeutics Announces Plan to Submit a New Drug Application for Accelerated Approval of Golodirsen in Patients with Duchenne Amenable to Skipping Exon 53


CAMBRIDGE, Mass., - Sarepta Therapeutics announced on March 12, 2018 that it recently received final minutes from a February 2018 Type C meeting held with the Division of Neurology Products, United States Food and Drug Administration (the Division), to solicit the Division's guidance on the development pathway for Sarepta's therapeutic candidate, golodirsen, a phosphordiamidate morpholino oligimer engineered to treat those patients with Duchenne muscular dystrophy (DMD) who have genetic mutations subject to skipping exon 53 of the DMD gene.

Please see the full release from Sarepta for more details. In the coming and weeks and months, we hope to learn more from Sarepta about what this guidance means for our patient community, especially those who may benefit from exon 53 skipping.