State and School Employees' Health Insurance Plan - Duchenne Muscular Dystrophy (DMD)

POLICY NUMBER

S.5.01.551

Amondys 45 (casimersen)

Exondys 51 (eteplirsen)

Elevidys (delandistrogene moxeparvovec-rokl)

Viltepso (viltolarsen)

Vyondys 53 (golodirsen)

Please perform a search of the State Health Plan Medical Drug Formulary for drugs administered and billed through the medical setting.

DESCRIPTION

Duchenne muscular dystrophy (DMD) is a genetic condition caused by mutations in the DMD gene. These mutations affect the dystrophin protein leading to progressive weakness and atrophy of heart and skeletal muscles.

Amondys 45 (casimersen) is indicated for the treatment of DMD in patients who have a confirmed mutation of the DMD gene that is amenable to exon 45 skipping.

Elevidys (delandistrogene moxeparvovec-rokl) is indicated for the treatment of DMD in patients at least 4 years of age who are ambulatory and have a confirmed mutation in the DMD gene.

Elevidys (delandistrogene moxeparvovec-rokl) is indicated for the treatment of DMD in patients who are non-ambulatory and have a confirmed mutation in the DMD gene. The DMD indication in non-ambulatory patients is approved under accelerated approval based on expression of ELEVIDYS micro-dystrophin. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).

Exondys 51 (eteplirsen) is indicated for treatment of DMD in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping.

Viltepso (viltolarsen) is indicated for the treatment of DMD in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping.

Vyondys 53 (golodirsen) is indicated for treatment of DMD in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping.

POLICY

A single-dose intravenous infusion of Elevidys (delandistrogene moxeparvovec-rokl) will be considered medically necessary when ALL of the following criteria are met:

1. The individual is >4 years of age at the time of infusion;

2. The individual has a confirmed mutation in the DMD gene;

3. The individual does not have any deletion in exon 8 and/or exon 9 in the DMD gene;

4. One day prior to infusion, a corticosteroid regimen for a minimum of 60 days will be initiated; AND

5. The prescribed dosage is within the program quantity limits based on FDA approved labeled dosage.

Length of Approval: 1 infusion per lifetime (weight based)

Elevidys (delandistrogene moxeparvovec-rokl) is considered investigational for the treatment of DMD in non-ambulatory individuals who have a confirmed mutation in the DMD gene as there is insufficient evidence of clinical benefit. 

Amondys 45 (casimersen), Exondys 51 (eteplirsen), Viltepso (viltolarsen), and Vyondys 53 (golodirsen) are considered investigational for the treatment of Duchenne muscular dystrophy (DMD) as there is insufficient evidence of clinical benefit.

Services related to delivery and/or administration of a medication determined to be not medically necessary will also be considered not medically necessary.

POLICY EXCEPTIONS

None

POLICY GUIDELINES

Medical Policy Manual coverage guidelines should not be used in lieu of the Participant's specific benefit plan language outlined in the Mississippi's State and School Employees’ Life and Health Insurance Plan.

Medically Necessary is defined as those services, treatments, procedures, equipment, drugs, devices, items or supplies furnished by a covered Provider that are required to identify or treat a Participant's illness, injury or Mental Health Disorders, and which Company determines are covered under this Benefit Plan based on the criteria as follows in A through D:

A. consistent with the symptoms or diagnosis and treatment of the Participant's condition, illness, or injury; and

B. appropriate with regard to standards of good medical practice; and

C. not solely for the convenience of the Participant, his or her Provider; and

D. the most appropriate supply or level of care which can safely be provided to the Participant. When applied to the care of an Inpatient, it further means that services for the Participant's medical symptoms or conditions require that the services cannot be safely provided to the Participant as an Outpatient.

For the definition of medical necessity, “standards of good medical practice” means standards that are based on credible scientific evidence published in peer-reviewed medical literature generally recognized by the relevant medical community, and physician specialty society recommendations, and the views of medical practitioners practicing in relevant clinical areas and any other relevant factors. BCBSMS makes no payment for services, treatments, procedures, equipment, drugs, devices, items or supplies which are not documented to be Medically Necessary. The fact that a Physician or other Provider has prescribed, ordered, recommended, or approved a service or supply does not in itself, make it Medically Necessary.

Investigative is defined as the use of any treatment procedure, facility, equipment, drug, device, or supply not yet recognized as a generally accepted standard of good medical practice for the treatment of the condition being treated and; therefore, is not considered medically necessary. For the definition of Investigative, “generally accepted standards of medical practice” means standards that are based on credible scientific evidence published in peer-reviewed medical literature generally recognized by the relevant medical community, and physician specialty society recommendations, and the views of medical practitioners practicing in relevant clinical areas and any other relevant factors. In order for equipment, devices, drugs or supplies [i.e, technologies], to be considered not investigative, the technology must have final approval from the appropriate governmental bodies, and scientific evidence must permit conclusions concerning the effect of the technology on health outcomes, and the technology must improve the net health outcome, and the technology must be as beneficial as any established alternative and the improvement must be attainable outside the testing/investigational setting.

POLICY HISTORY

07/01/2023: New policy added.

04/01/2024: Policy updated to state that Elevidys (delandistrogene moxeparvovec-rokl) is considered investigational for the treatment of Duchenne muscular dystrophy (DMD) as there is insufficient evidence of clinical benefit. Sources updated. Code Reference section updated to add HCPCS code J1413.

03/20/2025: Policy reviewed and approved by Pharmacy & Therapeutics (P&T) Committee. Policy description updated regarding indications for Elevidys (delandistrogene moxeparvovec-rokl). Policy section updated to create separate investigational and not medically necesssary policy statements for Elevidys (delandistrogene moxeparvovec-rokl). Sources updated.

07/01/2025: Policy updated to state that a single dose of Elevidys (delandistrogene moxeparvovec-rokl) will be considered medically necessary for the treatment of DMD in individuals who have a confirmed mutation in the DMD gene when criteria are met. Sources updated. J1413 moved from the Investigational Codes table to the Medically Necessary Codes table. Added G71.01 to the Medically Necessary Codes table.

SOURCE(S)

1. Amondys 45 prescribing information. Sarepta Therapeutics, Inc. July 2024. Last accessed July 2025.

2. Elevidys prescribing information. Sarepta Therapeutics, Inc. September 2024. Last accessed July 2025.

3. Exondys 51 prescribing information. Sarepta Therapeutics, Inc. January 2025. Last accessed July 2025.

4. National Center for Advancing Translational Sciences, Genetic and Rare Diseases Information Center, Duchenne Muscular Dystrophy. Available at:

   https://rarediseases.info.nih.gov/diseases/6291/duchenne-muscular-dystrophy

5. Viltepso prescribing information. NS Pharma, Inc. August 2023. Last accessed July 2025.

6. Vyondys 53 prescribing information. Sarepta Therapeutics, Inc. August 2020. Last accessed July 2025.

CODE REFERENCE

This may not be a comprehensive list of procedure codes applicable to this policy.

The code(s) listed below are ONLY medically necessary if the procedure is performed according to the "Policy" section of this document.

Medically Necessary Codes

Investigational Codes

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