WERE YOUR PATIENTS’ GENETIC TESTS PERFORMED USING A DIFFERENT METHOD? A NEW TEST IS REQUIRED TO ACCURATELY DETERMINE EXON 51 AMENABILITY.2

Exon Deletions Chart

 


 

WHICH MUTATIONS ARE AMENABLE TO EXON 51 SKIPPING?3-5

For exon skipping to work, exon 51 must be present in the patient's DMD gene. 

 

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EXPLORE THE DMD OPEN-ACCESS VARIANT EXPLORER (DOVE), A TOOL TO AGGREGATE CLINICALLY RELEVANT INFORMATION RELATED TO VARIANTS IN THE DMD GENE, AT DMD.NL/DOVE

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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. Aartsma-Rus A, Ginjaar IB, Bushby K. The importance of genetic testing for Duchenne muscular dystrophy. J Med Genet. 2016;0:1-7.
  3. den Dunnen JT. Leiden Muscular Dystrophy pages. Leiden, Netherlands: Center for Human and Clinical Genetics, Leiden University Medical Center; 2003. Available at dmd.nl. Accessed June 1, 2018.
  4. Fletcher S, Adams AM, Johnsen RD, et al. Mol. Ther. 2010;18(6): 1218-1223.
  5. Data on file.
  6. Aartsma-Rus A, et al. Theoretic applicability of anitsense-mediated exon skipping for Duchenne muscular dystrophy mutations. Human Mutation. 2009;X:1-7.