ModifiedBaylor Scott & White Health PlanPolicy 280

Gene Based Therapies for Duchenne Muscular Dystrophy

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This policy governs prior authorization and medical necessity criteria for gene-based and exon-skipping medications for Duchenne muscular dystrophy (DMD) for Baylor Scott & White Health Plan members, including casimersen, eteplirsen, golodirsen, viltolarsen, and delandistrogene moxeparvovec.

Policy Summary

PayerBaylor Scott & White Health Plan

PolicyGene Based Therapies for Duchenne Muscular Dystrophy

Policy CodePolicy 280

Change TypeAuthorization duration and title updates

Effective Date03/01/2026

Last ReviewFeb 26, 2026

Next Review Date02/26/2027

Key ActionSubmit prior authorization with diagnosis, genetic testing confirming mutation and amenability to exon skipping, required baseline labs, and functional testing (e.g., 6-minute walk test).

SourceLink

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ModifiedBaylor Scott & White Health PlanPolicy 280

Gene Based Therapies for Duchenne Muscular Dystrophy

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Sign up for Baylor Scott & White Health Plan Policy 280 alerts

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Policy Summary

PayerBaylor Scott & White Health Plan

PolicyGene Based Therapies for Duchenne Muscular Dystrophy

Policy CodePolicy 280

Change TypeAuthorization duration and title updates

Effective Date03/01/2026

Last ReviewFeb 26, 2026

Next Review Date02/26/2027

Key ActionSubmit prior authorization with diagnosis, genetic testing confirming mutation and amenability to exon skipping, required baseline labs, and functional testing (e.g., 6-minute walk test).

SourceLink

POLICY UPDATE CHANGES

Adjusted authorization duration to 12 months for casimersen, eteplirsen, golodirsen, and viltolarsen.

Renamed policy to 'Gene Based Therapies for Duchenne Muscular Dystrophy.'

5therapies named

>=300 m6MWT min (initial)

1delandistrogene lifetime

>=1:400Anti-AAVrh74 titer limit

12 moauthorization duration

Coverage Criteria for Gene-Based and Exon-Skipping Therapies

Casimersen (Amondys 45)

Covered when ALL of the following are met:

Casimersen criteria: 1. Member meets all universal criteria; AND 2. Treatment initiated before 14 years of age; AND 3. DMD gene mutation is amenable to exon 45 skipping (see Appendix A); AND 4. Initial authorization: member is able to achieve an average distance of at least 300 meters while walking independently over 6 minutes; AND 5. Renewal: within 6 months of request provide evidence of improvement, stabilization, or reduced decline (remaining ambulatory) AND manageable or no side effects.6MWT >= 300 m

Authorization duration: shortest of clinically appropriate, requested, or 12 months as applicable.

Eteplirsen (Exondys 51)

Coverage stance:

Eteplirsen (Exondys 51) for the treatment of Duchenne muscular dystrophy is considered not medically necessary as clinical benefit has not been established.

Use of eteplirsen for indications other than DMD is considered experimental and investigational.

Golodirsen (Vyondys 53)

Covered when ALL of the following are met:

Golodirsen criteria: 1. Member meets all universal criteria; AND 2. Treatment initiated before 16 years of age; AND 3. DMD gene mutation is amenable to exon 53 skipping (see Appendix C); AND 4. Initial authorization: member is able to achieve an average distance of at least 300 meters while walking independently over 6 minutes; AND 5. Renewal: within 6 months of request provide evidence of improvement, stabilization, or reduced decline (remaining ambulatory) AND manageable or no side effects.6MWT >= 300 m

Authorization duration: shortest of clinically appropriate, requested, or 12 months as applicable.

Viltolarsen (Viltepso)

Covered when ALL of the following are met:

Viltolarsen criteria: 1. Member meets all universal criteria; AND 2. Treatment initiated before 10 years of age; AND 3. DMD gene mutation is amenable to exon 53 skipping (see Appendix C); AND 4. Initial authorization: member is able to walk independently without assistive devices; AND 5. Renewal: within 6 months of request provide evidence of improvement, stabilization, or reduced decline (remaining ambulatory) AND manageable or no side effects.

Authorization duration: shortest of clinically appropriate, requested, or 12 months as applicable.

Delandistrogene moxeparvovec-rokl (Elevidys)

Covered when ALL of the following are met:

Delandistrogene criteria: 1. Member meets all universal criteria; AND 2. Member is 4 years of age or older; AND 3. Genetic testing conducted to confirm diagnosis and identify the specific DMD gene mutation; AND 4. Member is ambulatory (able to walk with or without assistance, not wheelchair dependent); AND 5. Baseline laboratory testing results for liver function, platelets, and troponin-1 are provided; AND 6. Member does NOT have any of the following: a) DMD gene deletion in exon 8 or exon 9; OR b) Anti-AAVrh74 total binding antibody titers ≥ 1:400; OR c) Concomitant exon skipping therapies for DMD; OR d) Current infection.anti-AAVrh74 <1:400

Only one lifetime administration is considered medically necessary; repeat administration is experimental/investigational. Authorization duration: shortest of clinically appropriate, requested, or as otherwise specified.

Delandistrogene (Elevidys) is contraindicated or not eligible for members with specific genetic or clinical findings. Members with a DMD gene deletion in exon 8 or exon 9 are excluded from treatment. Treatment is also not appropriate for members with anti-AAVrh74 total binding antibody titers ≥ 1:400. Active or current systemic infection at the time of planned administration is an exclusion. Additionally, delandistrogene must not be given concomitantly with other exon-skipping therapies for DMD (for example casimersen, eteplirsen, golodirsen, or viltolarsen).

Only a single lifetime administration of delandistrogene moxeparvovec-rokl is considered medically necessary. Repeat administration is experimental and investigational and is not covered because the effectiveness and safety of re-dosing have not been established.

No additional explicit exclusions beyond those stated in the policy text are listed in the cited document chunks. The policy references external literature and will be updated as new evidence emerges.

Eteplirsen (Exondys 51) is considered not medically necessary for the treatment of Duchenne muscular dystrophy because clinical benefit has not been established.

Use of the listed therapies — casimersen, eteplirsen, golodirsen, viltolarsen, and delandistrogene — for indications other than Duchenne muscular dystrophy is considered experimental and investigational and therefore not covered, as clinical effectiveness for other indications has not been established.

Within the provided document chunks there are no additional statements enumerating other specific conditions as 'not medically necessary.' The policy will continue to be revised based on emerging evidence and referenced literature as noted.

Coding — HCPCS, ICD-10, and Clinical Metrics

HCPCS Codes for DMD therapiesHCPCSCovered

J1428	Injection, eteplirsen (Exondys 51)
J1429	Injection, golodirsen (Vyondys 53)
J1427	Injection, viltolarsen (Viltepso)
J1426	Injection, casimersen (Amondys 45)

ICD-10 CodesICD-10

G71.0

Muscular dystrophy [Duchenne muscular dystrophy (DMD)]

6-Minute Walk Test (6MWT) — Initial Authorization Threshold

6-Minute Walk Test thresholdAverage distance ≥ 300 meters while walking independently (no side‑by‑side assist, cane, walker, wheelchair, etc.) over 6 minutes required for initial authorization of casimersen and golodirsen

Functional requirement for initial authorizationMember must be ambulatory and able to walk independently when performing the 6MWT

Documentation timing for renewalRenewal requires evidence within 6 months prior to request showing improvement, stabilization, or reduced decline (remaining ambulatory)

Provider Actions, Authorization, and Documentation

Prior Authorization

Prior Authorization Required

Prior authorization is required for all requests. Requests must demonstrate that the member meets the policy's universal and drug‑specific criteria and include supporting clinical documentation. All requests will be reviewed by a clinical pharmacist and medical director.

Prior authorization required — submit prior authorization request before therapy initiation.
Requests may be denied if the member has prior treatment with delandistrogene (Elevydis) or has anti‑AAVrh74 total binding antibody titers ≥ 1:400 where specified.
Requests may be denied if concomitant use of multiple exon‑skipping therapies is documented.
Authorization duration: up to the shortest of clinically appropriate duration, requested duration, or 12 months for casimersen, eteplirsen, golodirsen, and viltolarsen.

Documentation Required

Documentation Required — Diagnosis, Genetic Testing, Prescribing Clinician, and Baseline Data

Definitions and Therapy Types

Exon‑skipping Therapy (Antisense Oligonucleotides)

MechanismAntisense oligonucleotides (PMO class) that promote skipping of specific DMD exons to produce an internally truncated, functional dystrophin protein

ExamplesAgents include eteplirsen (Exondys 51), golodirsen (Vyondys 53), viltolarsen (Viltepso), and casimersen (Amondys 45)

Regulatory basisAccelerated approvals for some agents (golodirsen, viltolarsen, casimersen) are based on increases in the surrogate marker dystrophin production in skeletal muscle; clinical benefit remains variably established

Gene Transfer Therapy — Delandistrogene (Elevidys)

Vector and transgeneDelandistrogene (Elevidys) uses an AAVrh74 vector to deliver a micro‑dystrophin transgene

Antibody consideration

Background — Duchenne Muscular Dystrophy (DMD)

Duchenne muscular dystrophy (DMD) is an X-linked, recessive disorder caused by mutations in the dystrophin gene that lead to progressive skeletal muscle degeneration and loss of ambulation. There is no cure; current gene-based and exon-skipping therapies aim to increase dystrophin production as a surrogate marker to slow disease progression. Natural history typically involves early childhood onset of muscle weakness, progressive decline in ambulation during childhood or adolescence, and eventual loss of independent walking. Because therapies differ in mechanism and evidence (exon-skipping antisense oligonucleotides versus AAVrh74-mediated micro-dystrophin gene transfer), eligibility depends on genotype amenability, functional status, and safety considerations such as pre-existing anti-AAV antibodies.

Revision History

2020-10-22policy_created

Policy created (new policy).

2021-07-22updated

Added information for casimersen.

2021-10-28updated

Added coverage criteria, appendices, and updated codes.

Policy Summary

PayerBaylor Scott & White Health Plan

PolicyGene Based Therapies for Duchenne Muscular Dystrophy

Policy CodePolicy 280

Change TypeAuthorization duration and title updates

Effective Date03/01/2026

Last ReviewFeb 26, 2026

Next Review Date02/26/2027

Key ActionSubmit prior authorization with diagnosis, genetic testing confirming mutation and amenability to exon skipping, required baseline labs, and functional testing (e.g., 6-minute walk test).

SourceLink

On This Page

Provide the following documentation with the authorization request to prevent administrative denial: diagnosis documentation, genetic testing results confirming DMD and identifying the specific mutation, treating clinician specialty or consultation note, and regimen details that align with FDA labeling. For product‑specific initial and renewal requirements, include baseline ambulatory/6‑minute walk documentation, baseline labs for gene therapy, and evidence of no contraindicated findings (e.g., exon 8/9 deletions for delandistrogene, active infection).

Submit: clinical diagnosis of DMD from medical record and prescribing/consulting physician specialty.
Submit: genetic testing results to confirm diagnosis and identify mutation amenable to specific exon skipping (appendices referenced in policy).
Submit: documentation that drug will be dosed per FDA labeling and will not be used concomitantly with other exon‑skipping therapies.
For delandistrogene (Elevidys): submit baseline liver function, platelet, and troponin‑I results; confirm ambulatory status; confirm absence of anti‑AAVrh74 titers ≥ 1:400 and absence of exon 8 or 9 deletions.

Denial Risk

Denial Risk Conditions

Denial risk conditions to verify before submission: prior treatment with delandistrogene when policy prohibits repeat therapy, documented anti‑AAVrh74 total binding antibody titers at or above the exclusion threshold, genetic deletions that exclude eligibility (e.g., exon 8 or 9 for Elevidys), concomitant use of other exon‑skipping therapies, and current infection for gene therapy candidates.

Prior treatment with delandistrogene may preclude coverage for repeat administration — BSWHP considers only one lifetime treatment medically necessary for delandistrogene.
Anti‑AAVrh74 total binding antibody titers ≥ 1:400 are exclusionary for delandistrogene.
Genetic deletions in exon 8 or exon 9 are exclusionary for delandistrogene.
Concomitant use of exon‑skipping therapies (e.g., casimersen, eteplirsen, golodirsen, viltolarsen) is not permitted.

Note

Coverage Determination and Policy Governance

Coverage decisions are governed by this Baylor Scott & White Health Plan policy. Refer to the plan's EOC/SPD, Medicare NCD/LCDs, or Texas Medicaid TMPPM where applicable. If no applicable NCD/LCD exists, apply the criteria in this policy. Use the policy appendices to determine mutation amenability for exon skipping.

Coverage determinations require adherence to the policy's universal and drug‑specific criteria.
For Medicare and Medicaid members, confirm and apply any program‑specific mandates or coverage determinations.
See policy appendices for lists of mutations amenable to exon skipping (e.g., Appendix A, Appendix C).

Note

Provider Action — High Importance

Actionable provider steps (high importance): obtain and submit required diagnostic and genetic documents, confirm prescribing clinician specialty, confirm absence of exclusionary prior treatments or antibody titers, and attach baseline functional and laboratory measures where required to support initial and renewal requests.

Ensure prior authorization is obtained before starting therapy.
Attach genetic test report showing mutation amenable to the requested exon‑skipping therapy or gene therapy.
For initial golodirsen/viltolarsen requests, include documentation of ambulatory status or 6‑minute walk results as specified.
For delandistrogene requests, include baseline labs (liver function, platelets, troponin‑I) and antibody titer results.

Not recommended for patients with anti‑AAVrh74 total binding antibody titers ≥ 1:400 because pre‑existing antibodies may impede transgene expression

Administration limitsOnly one lifetime administration is considered medically necessary; repeat administration is experimental/investigational

Listed Therapies Included in This Policy

Therapies named in policyCasimersen (Amondys 45), Eteplirsen (Exondys 51), Golodirsen (Vyondys 53), Viltolarsen (Viltepso), Delandistrogene moxeparvovec‑rokl (Elevidys)

Therapy classesIncludes exon‑skipping antisense oligonucleotides and AAVrh74 vector gene transfer therapy

Indication scopeAll listed therapies are addressed for treatment of Duchenne muscular dystrophy; use for other indications is considered experimental/investigational

2022-10-28reviewed

Policy reviewed (no changes reported).

2023-09-28updated

Updated Medicaid instructions.

2024-11-11updated

Updated format and layout, separated universal criteria, added criteria and background for delandistrogene, updated authorization duration, and added requirement of RPh and medical director review.

2025-08-11updated

Removed Medicare NCD/LCD InterQual statement for clarity.

2025-11-10updated

Renamed policy to 'Gene Based Therapies for Duchenne Muscular Dystrophy,' updated universal request heading, standardized authorization duration language and investigational language, simplified background, and standardized references.

2026-02-26updatedLatest

Adjusted authorization duration to 12 months for casimersen, eteplirsen, golodirsen, and viltolarsen.