ACCELERATED APPROVAL BASED ON CLINICAL TRIAL RESULTS.
The FDA may grant Accelerated Approval based on clinical trials that:2
- are adequate and well controlled and
- establish the product has an effect on a surrogate endpoint that is reasonably likely to predict a clinical benefit.
Accelerated approval requires further adequate and well-controlled studies to verify and describe clinical benefit.2
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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. Code of Federal Regulations. 21 CFR 314. Available at: https://www.accessdata.fda.gov/scripts/cdrh/cfdocs/cfcfr/CFRSearch.cfm?CFRPart=314. Updated September 4,2018. Accessed November 29, 2018.
US Food and Drug Administration. Center Director Decisional Memo. Reference ID: 3959035. July 14, 2016.