WHA

AMONDYS 45 (casimersen)

Office Administration - intravenous

Diagnosis considered for coverage:
  • 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 45 skipping. This indication is approved under accelerated approval based on an increase in dystrophin production in skeletal muscle observed in patients treated with Amondys 45®. Continued approval for this indication may be contingent upon verification of a clinical benefit in confirmatory trials.
Coverage Criteria:

For diagnosis of Duchenne Muscular Dystrophy (DMD):

  • Dose does not exceed 30 mg/kg of body weight infused once weekly, AND
  • Prescribed by or in consultation with a neurologist with expertise in the treatment of DMD; AND
  • Submission of medical records (e.g., chart notes, laboratory values) documenting all of the following:
    • Diagnosis of Duchenne Muscular Dystrophy (DMD); AND
    • Documentation of a confirmed mutation of the dystrophin gene amenable to exon 45 skipping; AND
    • Member is ambulatory without assistance with a 6-minute walk test (6MWT) of at least 300 meters; AND
  • Member is between the ages of 7 to 13 years old at therapy initiation; AND
  • Currently on a stable dose or dose equivalent of oral corticosteroids for at least 6 months; AND
  • Amondys 45® is not prescribed concomitantly with other exon-skipping therapies (e.g., Exondys 51™, Vyondys 53™, Viltepso™)
Reauthorization Criteria:

For diagnosis of Duchenne Muscular Dystrophy (DMD):

  • Dose does not exceed 30 mg/kg of body weight infused once weekly; AND
  • Prescribed by or in consultation with a neurologist who has experience treating DMD; AND
  • Medical records (e.g., chart notes, laboratory values) documenting the member is maintaining ambulatory status, as evaluated via the 6-minute walk test (6MWT), AND
  • Currently on a stable dose or dose equivalent of oral corticosteroids; AND
  • Amondys 45® is not prescribed concomitantly with other exon-skipping therapies (e.g., Exondys 51™, Vyondys 53™, Viltepso™)
Coverage Duration:
  • Initial: 6 months
  • Reauthorization: 12 months
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:
  • Amondys 45® has not been studied in DMD that is not amenable to exon 45 skipping, or in other forms of muscular dystrophy.
  • Serum cystatin C, urine dipstick, and urine protein-to-creatinine ratio (UPCR) should be measured before starting Amondys 45®. Consider measurement of glomerular filtration rate prior to initiation of Amondys 45®. Monitoring for kidney toxicity during treatment is recommended. Obtain the urine sample prior to infusion of Amondys 45® or at least 48 hours after infusion.
  • If a persistent increase in serum cystatin C or proteinuria is detected, refer to a pediatric nephrologist for further evaluation.
Policy Updates:
  • 08/17/2021 – New policy approved by P&T.
References:
  • Amondys 45 [package insert], Cambridge, MA: Sarepta Therapeutics, Inc.; February 2021.
  • Birnkrant DJ, Bushby K, Bann CM, et al. Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and neuromuscular, rehabilitation, endocrine, and gastrointestinal and nutritional management. Lancet Neurol. 2018;17(3):251-267. doi: 10.1016/S1474-4422(18)30024-3.
  • Darras BT. Duchenne and Becker muscular dystrophy: Clinical features and diagnosis. UpToDate Web site. Updated July 24, 2020. https://www.uptodate.com/contents/duchenne-and-becker-muscular-dystrophy-clinical-features-and-diagnosis. Accessed April 19, 2021.
  • ClinicalTrials.gov Web site. https://clinicaltrials.gov. Accessed July 6, 2021.
  • Gloss D, Moxley RT, Ashwal S, Oskoui M. Practice guideline update summary: Corticosteroid treatment of Duchenne muscular dystrophy: Report of the Guideline Development Subcommittee of the American Academy of Neurology. Neurology. 2016;86(5):465-472. doi: 10.1212/WNL.0000000000002337.
  • McDonald CM, Henricson EK, Han JJ, et al. The 6-minute walk test as a new outcome measure in Duchenne muscular dystrophy. Muscle Nerve. 2010[b];41:500-510.
  • Messina S, Vita GL. Clinical management of Duchenne muscular dystrophy: the state of the art. Neurol Sci. 2018;39(11):1837-1845. doi: 10.1007/s10072-018-3555-3.

 

 

Last review date: August 17, 2021