Recommend the addition of delandistrogene moxeparvovec-rokl (Elevidys) to OH Epic formulary, available via a therapy plan, with the following criteria of use:

• Indication: Duchenne muscular dystrophy (DMD), ambulatory, with a confirmed mutation in the DMD gene.
• Service line: Pediatric Neurology
• Formulary location: Outpatient (NOMH)
• Service location: Outpatient infusion (NOMH)
• Patient population: Pediatrics aged ≥ 4 years
• Prior authorization required: Yes—via single case agreement
• Restriction/criteria of use (all will apply unless specified):
  • Authorization for treatment will be subject to single case agreement confirmation
  • Diagnosis of Duchenne muscular dystrophy mutation with a confirmed mutation in the DMD gene
  • Patient must be ambulatory and without cardiomyopathy
  • Prescribed by or in consultation with a specialist (e.g., pediatric neurologist, neuromuscular specialist) with experience in the treatment of DMD
  • Patient does not have a deletion in exon 8 or exon 9 of the DMD gene
  • Patient does not have deletions in exon 1 to 17 or exon 61 to 79
  • Patient does not have an elevated anti-AAVrh74 total binding antibody titer ≥ 1:400
  • Patient will receive a corticosteroid regimen prior to and following receipt of Elevidys
  • Patient will not receive exon-skipping therapies for DMD concomitantly or following Elevidys treatment
  • Patient does not have an active infection
  • Patient does not have significant liver dysfunction or disease (e.g., pre-existing liver impairment, chronic hepatitis)
  • Patient has never received Elevidys treatment in their lifetime
  • Elevidys dosing is in accordance with FDA approved labeling
  • Authorization will be issued for no more than one treatment per lifetime

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