Exondys 51 Packaging

EXONDYS 51 is indicated for patients with Duchenne who have a confirmed mutation in 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.1

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STUDY RESULTS SHOW INCREASE IN DYSTROPHIN IN SOME EXONDYS 51 PATIENTS.1

Dystrophin was quantified by a validated, reliable and reproducible Western blot assay (Week 180 for Study 2 and Week 48 for Study 3).

 

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APPROVED UNDER FDA ACCELERATED APPROVAL.1

Accelerated approval applies to new medicines that have been studied for safety and effectiveness in treating serious or life-threatening illnesses and provide meaningful therapeutic benefit to patients over existing treatments.2

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SURROGATE ENDPOINT.

The surrogate endpoint in the analysis for accelerated approval of EXONDYS 51 was expression of dystrophin protein. An FDA analysis concluded that an increase in dystrophin in skeletal muscle in some patients treated with EXONDYS 51 was reasonably likely to predict a clinical benefit.1,3

 

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INDICATION

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.

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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.

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.

In the 88 patients who received ≥30 mg/kg/week of EXONDYS 51 for up to 208 weeks in clinical studies, the following events were reported in ≥10% of patients and occurred more frequently than on the same dose in Study 1: vomiting, contusion, excoriation, arthralgia, rash, catheter site pain, and upper respiratory tract infection.

Please see the full Prescribing Information for EXONDYS 51 (eteplirsen).

 

 

References

  1. EXONDYS 51 [package insert]. Cambridge, MA: Sarepta Therapeutics Inc; October 2018.
  2. 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.
  3. US Food and Drug Administration. Center Director Decisional Memo. Reference ID: 3959035. July 14, 2016.