Background

Duchenne muscular dystrophy (DMD) is a progressive, X-linked, degenerative neuromuscular disease that results in disabling muscle weakness and eventually leads to early death. DMD is caused by mutations in the dystrophin gene resulting in reduced or near absence of dystrophin, a protein that helps keep muscle cells intact. The estimated incidence of DMD is 1 in 3500–5000 male births (i.e., 400 to 600 boys per year) with prevalence estimates ranging between 10,000 and 15,000 males.

Delandistrogene moxeparvovec-rokl (e.g., Elevidys) is an adeno-associated virus vector-based gene therapy indicated for the treatment of ambulatory pediatric individuals aged 4 through 5 years with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene.

Policy

The use of Elevidys is considered INVESTIGATIONAL/UNPROVEN for all indications including treatment of Duchenne muscular dystrophy (DMD).

References

1. Elevidys [package insert]. Cambridge (MA): Sarepta Therapeutics, Inc. 2023
2. Birnkrant DJ, Bushby K, Bann CM, et al.(2018) Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and neuromuscular, rehabilitation, endocrine, and gastrointestinal and nutritional management. Lancet Neurol. Mar 2018; 17(3): 251-267. PMID 29395989
3. Birnkrant DJ, Bushby K, Bann CM, et al.(2018) Diagnosis and management of Duchenne muscular dystrophy, part 2: respiratory, cardiac, bone health, and orthopaedic management. Lancet Neurol. Apr 2018; 17(4): 347-361. PMID 29395990
4. Bushby K, Finkel R, Birnkrant DJ, et al.(2010) Diagnosis and management of Duchenne muscular dystrophy, part 2: implementation of multidisciplinary care. Lancet Neurol. Feb 2010; 9(2): 177-89. PMID 19945914
5. Center for Disease Control and Prevention. Muscular Dystrophy: MD STARnet Data and Statistics. Available at http://www.cdc.gov/ncbddd/musculardystrophy/data.html. Accessed July 7, 2023.
6. Duchenne Muscular Dystrophy and Related Dystrophinopathies: Developing Drugs for Treatment Guidance for Industry. Published February 2018. Available at https://www.fda.gov/media/92233/download. Accessed July 7, 2023.
7. Falzarano MS, Scotton C, Passarelli C, et al.(2015) Duchenne Muscular Dystrophy: From Diagnosis to Therapy. Molecules. Oct 07 2015; 20(10): 18168-84. PMID 26457695
8. Feingold B, Mahle WT, Auerbach S, et al.(2017) Management of Cardiac Involvement Associated With Neuromuscular Diseases: A Scientific Statement From the American HearAssociation. Circulation. Sep 26 2017; 136(13): e200-e231. PMID 28838934
9. Food and Drug Administration.(2023) Sponsor Briefing Document for SRP-9001 (delandistrogene moxeparvovec-rokl) for the treatment of duchenne muscular dystrophy. Cellular, Tissue, and Gene Therapies Advisory Committee. Meeting date 12 May 2023. Available at https://www.fda.gov/media/168022/download. Accessed July 12, 2023.
10. Food and Drug Administration.(2023) Summary Basis for Regulatory Action- Elevidys. June 21, 2023. Available at https://www.fda.gov/media/169746/download. Accessed July 5, 2023.
11. Gloss D, Moxley RT, Ashwal S, et al.(2016) Practice guideline update summary: Corticosteroid treatment of Duchenne muscular dystrophy: Report of the Guideline Development Subcommittee of the American Academy of Neurology. Neurology. Feb 02 2016; 86(5): 465-72. PMID 26833937
12. Henricson E, Abresch R, Han JJ, et al.(2013) The 6-Minute Walk Test and Person-Reported Outcomes in Boys with Duchenne Muscular Dystrophy and Typically Developing Controls: Longitudinal Comparisons and Clinically-Meaningful Changes Over One Year. . PLoS Curr. Jul 08 2013; 5. PMID 23867975
13. Klimchak AC, Sedita LE, Rodino-Klapac LR, Mendell JR, et al.(2023) Assessing the value of delandistrogene moxeparvovec-rokl (SRP-9001) gene therapy in patients with Duchenne muscular dystrophy in the United States. J Mark Access Health Policy. 2023 May 26;11(1):2216518. doi: 10.1080/20016689.2023.2216518. PMID: 37261034; PMCID: PMC10228300
14. McDonald CM, Henricson EK, Abresch RT, et al.(2013) The 6-minute walk test and other endpoints in Duchenne muscular dystrophy: longitudinal natural history observations over 48 weeks from a multicenter study. Muscle Nerve. Sep 2013; 48(3): 343-56. PMID 23681930
15. Muscular Dystrophy Association (MDA). Available at mda.org/disease/Duchenne-musculardystrophy. Last accessed July 24, 2023. Prescribing Label: Elevidys (delandistrogene moxeparvovec-rokl-rokl) suspension, for intravenous infusion. Available at https://www.fda.gov/media/169679/download. Accessed on July 5, 2023.
16. Zambon AA, Ayyar Gupta V, Ridout D, et al.(2022) Peak functional ability and age at loss of ambulation in Duchenne muscular dystrophy. Dev Med Child Neurol. Aug 2022; 64(8): 979-988. PMID 35385138.

Coding Section

Procedure and diagnosis codes on Medical Policy documents are included only as a general reference tool for each policy. They may not be all-inclusive.

This medical policy was developed through consideration of peer-reviewed medical literature generally recognized by the relevant medical community, U.S. FDA approval status, nationally accepted standards of medical practice and accepted standards of medical practice in this community, and other nonaffiliated technology evaluation centers, reference to federal regulations, other plan medical policies and accredited national guidelines.

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