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Antisense Oligonucleotide Therapy for Duchenne Muscular Dystrophy

Commercial Drug Policy
Version Date: October 2021

Restricted Product(s):

  • casimersen (Amondys 45®) intravenous infusion for administration by a healthcare professional
  • eteplirsen (Exondys 51®) intravenous infusion for administration by a healthcare professional
  • golodirsen (Vyondys 53®) intravenous infusion for administration by a healthcare professional
  • viltolarsen (Viltepso®) intravenous infusion for administration by a healthcare professional

FDA Approved Use:

  • Casimersen (Amondys 45®)
    • For treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 45 skipping*
  • Eteplirsen (Exondys 51®)
    • For treatment of DMD in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping*
  • Golodirsen (Vyondys 53®)
    • For treatment of DMD in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping*
  • Viltolarsen (Viltepso®)
    • For treatment of DMD in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping*

*This indication is approved under accelerated approval based on an increase in dystrophin production in skeletal muscle observed in patients treated with above antisense oligonucleotide therapies. Continued FDA approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.

Criteria for Medical Necessity:

Not applicable

The use of antisense oligonucleotide therapy (i.e., casimersen [Amondys 45], eteplirsen [Exondys 51], golodirsen [Vyondys 53], and viltolarsen [Viltepso]) are considered investigational for all indications including treatment of Duchenne muscular dystrophy. BCBSNC does not provide coverage for investigational services or procedures.

  • The use of casimersen is considered investigational for all indications including treatment of Duchenne muscular dystrophy
  • The use of eteplirsen is considered investigational for all indications including treatment of Duchenne muscular dystrophy
  • The use of golodirsen is considered investigational for all indications including treatment of Duchenne muscular dystrophy
  • The use of viltolarsen is considered investigational for all indications including treatment of Duchenne muscular dystrophy

FDA Label Reference

MedicationIndicationDosingHCPCSMaximum Units
casimersen (Amondys 45®)
intravenous (IV) infusion		DMD in patients who have a confirmed variant of the DMD gene that is amenable to exon 45 skipping30 mg/kg IV once weeklyJ1426N/A
eteplirsen (Exondys 51®)
intravenous (IV) infusion		DMD in patients who have a confirmed variant of the DMD gene that is amenable to exon 51 skipping30 mg/kg IV once weeklyJ1428N/A
golodirsen (Vyondys 53®)
intravenous (IV) infusion		DMD patients who have a confirmed variant of the DMD gene that is amenable to exon 53 skipping30 mg/kg IV once weeklyJ1429N/A
viltolarsen (Viltepso®)
intravenous (IV) infusion		DMD patients who have a confirmed variant of the DMD gene that is amenable to exon 53 skipping80 mg/kg IV once weeklyJ1427N/A

References:

All information referenced is from FDA package insert unless otherwise noted below.

Policy Implementation/Update Information:

October 2021: Coding update: Added HCPCS code J1426 to dosing reference table effective 10/1/2021, deleted C9075, J3490, and J3590 termed 9/30/2021.

July 2021: Coding update: Added HCPCS code C9075 to dosing reference table effective 7/1/2021, deleted C9399 termed 6/30/2021.

June 2021: Criteria change: Updated medical policy formatting. Policy notification given 4/16/2021 for effective date 6/16/2021.

*Further historical criteria changes and updates available upon request from Medical Policy and/or Corporate Pharmacy.

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