CurrentCapital BluecrossPolicy MP 2.382

Treatment for Duchenne muscular dystrophy (antisense oligonucleotides)

This policy addresses coverage stance for antisense oligonucleotide therapies (eteplirsen, golodirsen, viltolarsen, casimersen) for Duchenne muscular dystrophy and applies to Capital Blue Cross products (with program/product variations noted).

Policy Summary

PayerCapital Bluecross

PolicyTreatment for Duchenne muscular dystrophy (antisense oligonucleotides)

Policy CodePolicy MP 2.382

Change TypeNo material changes

Effective Date

Next Review Date

Key ActionVerify member benefits and preauthorization requirements with the member's health benefit plan before proceeding.

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

4named antisense therapies

≈0.4–5.3%mean dystrophin increase

45,51,53exon targets referenced

Listedinvestigational codes

29+references

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Coverage Determination and Evidence Summary

Overall coverage determination

Covered when ALL of the following are met:

Investigational stance: Use of antisense oligonucleotides (eteplirsen, golodirsen, viltolarsen, casimersen) is considered investigational for all indications

Insufficient evidence to support health outcomes or benefits.

Evidence conclusions for exon-skipping therapies

Summary conclusions on evidence for exon‑skipping therapies:

Viltolarsen evidence conclusion: For individuals with DMD amenable to exon 53 skipping who receive viltolarsen, current evidence (a 2‑part multicenter study including an 80 mg/kg weekly IV cohort) demonstrates increases in dystrophin expression (mean increase 5.3% of normal at week 25) but lacks satisfactory data that truncated dystrophin confers clinical benefit; a confirmatory, prospective, adequately powered trial is necessary.mean dystrophin increase 5.3% at week 25

Evidence insufficient to determine improvement in net health outcome

Casimersen evidence conclusion: For individuals with DMD amenable to exon 45 skipping who receive casimersen, interim phase 3 data show small but statistically significant increases in dystrophin (mean change 0.81% vs 0.22% at week 48; between‑arm difference 0.59%), however there are no satisfactory data establishing that this level of dystrophin translates to meaningful clinical benefit; confirmatory trials are necessary.mean dystrophin increase 0.59% at week 48

Evidence insufficient to determine improvement in net health outcome

General statement on eteplirsen/golodirsen evidence: Reviews (e.g., ICER) conclude that increases in dystrophin with eteplirsen and golodirsen are of uncertain clinical or biological importance and that high‑ or moderate‑quality evidence demonstrating functional improvements is lacking; patient‑important outcome data are limited.NA

Certainty of net benefit is low

The policy designates the use of antisense oligonucleotides (including eteplirsen, golodirsen, viltolarsen, and casimersen) as investigational for all indications. As stated in the policy, these products are excluded from coverage because current evidence is insufficient to support a general conclusion regarding their health outcomes or clinical benefit.

Specific procedure/drug billing codes identified in the policy are listed as investigational and therefore not covered. Examples included in the investigational section are HCPCS codes J1426, J1427, J1428, and J1429.

No additional explicit coverage exclusions beyond the investigational designation and associated investigational codes are stated in the cited policy history or administrative notes.

For exon‑skipping therapies (for example, viltolarsen for exon 53 and casimersen for exon 45), clinical studies report increases in dystrophin expression (e.g., mean increase ~5.3% for viltolarsen at week 25 and a mean difference of ~0.59%–0.81% for casimersen at week 48). However, the policy concludes that available evidence is insufficient to determine an improvement in net health outcome because the clinical relevance of these dystrophin increases is unestablished and confirmatory, adequately powered trials demonstrating patient‑important functional benefit are lacking.

The cited policy sections do not specify any conditions labeled explicitly as 'not medically necessary' beyond the investigational determination; no separate NMN criteria are provided in these chunks.

Billing and Diagnosis Codes

Investigational HCPCS CodesHCPCSNot Covered

J1426	listed under Investigational, therefore not covered
J1427	listed under Investigational, therefore not covered
J1428	listed under Investigational, therefore not covered
J1429	listed under Investigational, therefore not covered

Diagnosis CodesICD-10

G71.00	Muscular dystrophy
G71.01	Duchenne or Becker muscular dystrophy
Z92.86	Personal history of gene therapy

Dystrophin increase reported (trial biomarker results summary)

Viltolarsen (Viltepso) mean dystrophin increaseMean increase 5.3% (±4.5) of normal levels at week 25 in 8 patients receiving 80 mg/kg weekly IV

Casimersen (Amondys45) mean dystrophin increaseMean change from baseline to week 48: 0.81% for casimersen versus 0.22% for placebo (interim analysis); reported 0.59% between-group increase at week 48

Clinical significanceDocumented increases in dystrophin expression exist but minimum beneficial level and translation to clinical benefit are not established

Provider Requirements, Prior Authorization, and Billing Guidance

Prior Authorization

Program/product variations

This policy applies only to certain Capital Blue Cross programs/products — benefit coverage varies by product. Before rendering a coverage determination or submitting a claim, verify the member's specific plan benefits and any FEP PPO variations (see the FEP Medical Policy Manual).

Program/product variations — check applicable benefits (e.g., FEP PPO may follow FEP Medical Policy Manual).
Confirm member-level benefits and preauthorization requirements with Capital Blue Cross Provider Services or Member Services.

Billing Rule

Codes applied at policy creation

The codes listed in the policy were applied at policy creation and are intended for informational/billing use only. Identification of a code here does not denote coverage; coverage is determined by the member's benefit plan.

Clinical Background

Duchenne muscular dystrophy (DMD) is an X‑linked, recessive neuromuscular disorder that primarily affects males and is caused by variants in the dystrophin gene leading to progressive muscle degeneration, loss of ambulation, and progressive respiratory and cardiac complications. Exon‑skipping antisense oligonucleotide therapies (such as eteplirsen, golodirsen, viltolarsen, and casimersen) are designed to skip specific exons in dystrophin mRNA to produce a truncated dystrophin protein; some have received FDA approval under accelerated pathways based on increases in dystrophin, but this policy considers their use investigational and does not find sufficient evidence of clinical benefit.

Therapies and Guideline References

Exon-skipping antisense oligonucleotide therapies

Eteplirsen (Exondys 51)Approved for DMD amenable to exon 51 skipping; accelerated approval based on increased dystrophin in skeletal muscle

Golodirsen (Vyondys 53)Approved for DMD amenable to exon 53 skipping; accelerated approval based on increased dystrophin in skeletal muscle

Viltolarsen (Viltepso)Approved for DMD amenable to exon 53 skipping; accelerated approval based on increased dystrophin production in skeletal muscle

Casimersen (Amondys45)Approved for DMD amenable to exon 45 skipping; accelerated approval based on increased dystrophin production in skeletal muscle

Exon-skipping therapy (viltolarsen, casimersen) — exon targets

Viltolarsen exon targetExon 53 skipping (amenable variants)

Policy Summary

PayerCapital Bluecross

PolicyTreatment for Duchenne muscular dystrophy (antisense oligonucleotides)

Policy CodePolicy MP 2.382

Change TypeNo material changes

Effective Date

Next Review Date

Key ActionVerify member benefits and preauthorization requirements with the member's health benefit plan before proceeding.

SourceLink

On This Page

Coding information provided in Section VII. Codes are subject to change and may not be all-inclusive.
Examples from the policy: procedure and J‑codes listed as investigational (see policy coding section).

Denial Risk

Investigational designation — denial risk

The policy designates antisense oligonucleotides for Duchenne muscular dystrophy as investigational for all indications. An investigational designation may be a basis for claim denial if the member's benefit plan excludes investigational services.

Investigational designation — potential denial trigger: services classified as investigational may be denied per plan exclusions.

Denial Risk

Investigational coding — denial risk

Certain codes included in the coding section are explicitly labeled investigational and therefore not covered; submitting those codes may result in denial.

Investigational / not covered codes noted in policy coding section (e.g., the listed J‑codes and associated procedure codes).
Codes are illustrative and may be incomplete; always verify current code sets before billing.

Note

No additional explicit denial triggers noted

The policy text does not list additional explicit provider documentation requirements or other specific denial triggers beyond the investigational designation and benefit exclusion language. Providers should still supply standard documentation to support medical necessity when requesting preauthorization.

No explicit additional denial triggers are present in the policy text beyond investigational designation and benefit-variation statements.
Provide clinical documentation (diagnosis, genetic testing results, prior therapies) when seeking preauthorization where applicable.

Prior Authorization

Confirm benefits and contact information

Before initiating treatment or submitting a preauthorization request, confirm member benefits, medical necessity requirements, and obtain any required prior authorization. Contact Capital Blue Cross Provider Services or Member Services for plan-specific guidance.

Confirm benefits and preauthorization requirements with Capital Blue Cross Provider Services or Member Services.
For FEP PPO members, refer to the FEP Medical Policy Manual at the FEP Blue website for applicable guidance.

Casimersen exon targetExon 45 skipping (amenable variants)

Other exon targets referencedEteplirsen targets exon 51; golodirsen targets exon 53

Corticosteroid treatment guideline reference

AAN corticosteroid guidelinePractice guideline update summary: Corticosteroid treatment of Duchenne muscular dystrophy; Neurology. Feb 02, 2016; 86(5): 465-72 (Gloss et al.)

PurposeGuideline provides recommendations on corticosteroid use in DMD management

Reference PMIDPMID 26833937

BCBSA policy reference for Duchenne treatment

BCBSA policy referenceBlue Cross Blue Shield Association Medical Policy Reference Manual. 5.01.27, Treatment for Duchenne Muscular Dystrophy. June 2022

Policy adoption notePolicy history indicates BCBSA full adoption referenced in policy history

Use in contextBCBSA policy cited as a reference supporting the policy's evidence and coverage stance