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
SURROGATE ENDPOINT: EXPRESSION OF DYSTROPHIN PROTEIN.
An FDA analysis of dystrophin data from patients treated with EXONDYS 51 concluded an increase in dystrophin in skeletal muscle in some patients was reasonably likely to predict a clinical benefit.3
DYSTROPHIN WAS PRODUCED IN SOME PATIENTS AS MEASURED BY SAREPTA WESTERN BLOT.1
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EXONDYS 51 (eteplirsen) 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. Continued approval for this indication may be contingent upon verification of a clinical benefit in confirmatory trials.
IMPORTANT SAFETY INFORMATION
Hypersensitivity reactions, including bronchospasm, chest pain, cough, tachycardia and urticaria, 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.
Adverse reactions in DMD patients (N=8) treated with EXONDYS 51 30 or 50 mg/kg/week by intravenous (IV) infusion with an incidence of at least 25% more than placebo (N=4) (Study 1, 24 weeks) were (EXONDYS 51, placebo): balance disorder (38%, 0%), vomiting (38%, 0%) and contact dermatitis (25%, 0%). The most common adverse reactions were balance disorder and vomiting. Because of the small numbers of patients, these represent crude frequencies that may not reflect the frequencies observed in practice. The 50 mg/kg once weekly dosing regimen of EXONDYS 51 is not recommended.
The most common adverse reactions from observational clinical studies (N=163) seen in greater than 10% of patients were headache, cough, rash, and vomiting.
Please see the full Prescribing Information for EXONDYS 51 (eteplirsen).
EXONDYS 51 [package insert]. Cambridge, MA: Sarepta Therapeutics Inc.
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 February 15, 2022.
US Food and Drug Administration. Center Director Decisional Memo. Reference ID: 3959035. July 14, 2016.