CLINICAL TRIAL RESULTS SHOW AN INCREASE IN DYSTROPHIN. 1,4,5
EXONDYS 51 uses exon skipping to address DMD. Clinical trials confirm the exon-skipping mechanism of action (MoA) of EXONDYS 51.4,5
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EXONDYS 51 is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping. This indication is approved under accelerated approval based on an increase in dystrophin in skeletal muscle 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.
IMPORTANT SAFETY INFORMATION
Hypersensitivity reactions, including rash and urticaria, pyrexia, flushing, cough, dyspnea, bronchospasm, and hypotension, have occurred in patients who were treated with EXONDYS 51. If a hypersensitivity reaction occurs, institute appropriate medical treatment and consider slowing the infusion or interrupting the EXONDYS 51 therapy.
EXONDYS 51 [package insert]. Cambridge, MA: Sarepta Therapeutics Inc; October 2018.
US Food and Drug Administration. FDA grants accelerate approval to first drug for Duchenne muscular dystrophy. https://www.fda.gov/newsevents/newsroom/pressannouncements/ucm521263.htm. Published September, 2016. Accessed June 1, 2018.
National Institutes of Health. National Library of Medicine. Genetics Home Reference. “Duchenne and Becker Muscular Dystrophy.” http://ghr.nlm.nih.gov/condition/duchenne-and-becker-muscular-dystrophy. Reviewed November, 2016. Accessed June 1, 2018.
Schnell FJ et al. Development of a Validated Western Blot Method for Quantification of Human Dystrophin Protein. Poster presented at the 21st International Congress of the World Muscle Society; October 2016; Granada Spain.
Data on file.