Duchenne Muscular Dystrophy (DMD)

POLICY NUMBER

L.5.01.551

Amondys 45 (casimersen)

Elevidys (delandistrogene moxeparvovec-rokl)

Exondys 51 (eteplirsen)

Viltepso (viltolarsen)

Vyondys 53 (golodirsen)

Please perform a formulary drug search on your patient’s member ID to ensure the prescription drug is covered under their benefit plan. The medication(s) in this medical policy may not be covered under a specific member’s benefit plan.

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 individuals at least 4 years of age who are ambulatory or non-ambulatory and have a confirmed mutation in the DMD gene.

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

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.

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.

Elevidys (delandistrogene moxeparvovec-rokl) is considered not medically necessary for the treatment of DMD in ambulatory individuals who have a confirmed mutation in the DMD gene as there is insufficient evidence to demonstrate clinical efficacy.

Services related to delivery and/or administration of a medication which have not been approved through the BCBSMS PA review process will be considered not medically necessary.

POLICY EXCEPTIONS

State Health Plan (State and School Employees) Participants

POLICY GUIDELINES

The coverage guidelines outlined in the Medical Policy Manual should not be used in lieu of the Member's specific benefit plan language.

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 Member'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:

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

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

3. not solely for the convenience of the Member, his or her Provider; and

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

04/15/2020: New policy added.

06/29/2020: Code Reference section updated to add new HCPCS code J1429, effective 07/01/2020.

02/18/2021: Added Viltepso (viltolarsen) to the top of the policy. Policy description updated to add indications for Viltepso (viltolarsen). Policy statement revised to include Viltepso (viltolarsen) as investigational for the treatment of Duchenne muscular dystrophy. Added statement that services related to delivery and/or administration of a medication which have not been approved through the BCBSMS PA review process will be considered not medically necessary. Policy Guidelines updated to change "Nervous/Mental Conditions" to "Mental Health Disorders" and to define investigative. Sources updated. Code Reference section updated to add HCPCS code C9071, J1428, and J3490.

03/25/2021: Code Reference section updated to add new HCPCS code J1427, effective 04/01/2021.

08/17/2021: Added Amondys 45 (casimersen) to the top of the policy. Policy description updated to add indication for Amondys 45 (casimersen). Investigational statement updated to include Amondys 45 (casimersen). Sources updated. Code Reference section updated to add HCPCS code C9075.

10/01/2021: Code Reference section updated to add new HCPCS code J1426, effective 10/01/2021.

02/01/2023: Policy Exceptions updated to add the following: State Health Plan (State and School Employees): The prescription drug(s) in this medical policy may be covered under a prescription drug benefit plan administered by the State Health Plan’s Pharmacy Benefit Manager. Please perform a formulary drug search at https://www.dfa.ms.gov/cvs-caremark and submit any required Prior Authorization Requests for coverage determination to the Plan’s Pharmacy Benefit Manager. Services related to delivery and/or administration of a medication determined to be not medically necessary will also be considered not medically necessary. Services related to delivery and/or administration of a self-administered drug are not covered.

07/01/2023: Policy Exceptions updated regarding State Health Plan (State and School Employees) Participants.

07/10/2023: Code Reference section updated to remove deleted HCPCS codes C9071 and C9075.

04/01/2024: Policy updated to add Elevidys (delandistrogene moxeparvovec-rokl) as 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.

12/11/2024: Policy description updated to add indications for Agamree (vamorolone). Policy statement updated to state that Agamree (vamorolone) is considered investigational for the treatment of Duchenne muscular dystrophy (DMD) as there is insufficient evidence of clinical benefit. Sources updated.

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

SOURCE(S)

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

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

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

6. Vyondys 53 prescribing information. Sarepta Therapeutics, Inc. August 2020. Last accessed February 2021.

CODE REFERENCE

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

Investigational Codes

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