ELEVIDYS (delandistrogene moxeparvovec-rokl)

ELEVIDYS (delandistrogene moxeparvovec-rokl)

Office-Administration – intravenous infusion

Diagnosis considered for coverage:

• Duchenne Muscular Dystrophy (DMD): Indicated for the treatment of ambulatory pediatric patients aged 4 through 5 years with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene. 
  • This indication is approved under accelerated approval based on expression of ELEVIDYS micro-dystrophin observed in patients treated with Elevidys. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).

Coverage Criteria:

For diagnosis of Duchenne muscular dystrophy (DMD):

• Diagnosis of Duchenne muscular dystrophy (DMD); AND
• Mutation in the DMD gene has been confirmed; AND
• No deletion in exon 8 or exon 9 in the DMD gene is present; AND
• Patient is 4 or 5 years of age; AND
• Patient is ambulatory without needing an assistive device (e.g., without side-by-side assist, cane, walker, wheelchair, etc.); AND
• Anti-AAVrh74 total binding antibody titers are less than 1:400; AND
• Patient will receive a corticosteroid regimen prior to and following the administration of Elevidys in line with the FDA-approved recommendations in the labeling; AND
• Provider attests that all of the following laboratory values have been checked prior to therapy and will be monitored after administration according to the FDA-approved recommendations in the labeling:
  • Liver function (i.e., clinical exam, GGT, total bilirubin) 
  • Platelet counts 
  • Troponin-I; AND
• Patient does not have clinical signs or symptoms of infection; AND
• Patient will not receive exon-skipping therapies for DMD [e.g., Amondys (casimersen), Exondys 51 (eteplirsen), Viltepso (viltolarsen), Vyondys 53 (golodirsen)] concomitantly or following Elevidys treatment; AND
• Prescribed by or in consultation with a neurologist who has experience treating children; AND
• Patient has never received Elevidys treatment in their lifetime.

Dosing:

DMD:

• The recommended dose of ELEVIDYS is 1.33 × 10¹⁴ vector genomes per kilogram (vg/kg) of body weight (or 10 mL/kg body weight)
  • ELEVIDYS dose (in mL) = patient body weight (in kilogram) x 10
  • Number of ELEVIDYS vials needed = ELEVIDYS dose (in mL) divided by 10 (round to the nearest number of vials)

Coverage Duration: 

• One (1) Time Authorization in Lifetime

Authorization is not covered for the following:

The use of this drug for indications not listed in this policy does not meet the coverage criteria established by the Western Health Advantage (WHA) Pharmacy and Therapeutics (P&T) Committee.

Additional Information: 

• DMD is the most common pediatric muscular dystrophy, with an incidence of around 400 to 600 cases per year and a prevalence of approximately 9,000 to 12,000 males in the United States (U.S.) (ICER 2019, Klimchak 2023). The estimated incidence worldwide is 1 in 3,500 to 5,000 live male births (Mendell et al 2013).
• Elevidys is a recombinant adeno-associated viral serotype rh74 (AAVrh74)-based gene therapy designed to deliver a copy of the gene encoding a micro-dystrophin protein expressed in normal muscle cells.
• Elevidys is contraindicated in patients with any deletion in exon 8 and/or exon 9 in the DMD gene.

Policy Updates:

• 03/01/2024 – New policy approved by WHA P&T Committee. (P&T, 02/20/2024)

References:

• Elevidys Prescribing Information. Sarepta Therapeutics, Inc. Cambridge, MA. June 2023.