ModifiedPremera BluecrossPolicy 5.01.570

Pharmacologic Treatment of Duchenne Muscular Dystrophy

Medical and pharmacy benefit coverage and site-of-service medical necessity criteria for pharmacologic therapies used to treat Duchenne muscular dystrophy (DMD), including exon-skipping antisense oligonucleotides, gene therapy, corticosteroids, and other specified agents. Affects providers requesting medical or pharmacy benefit authorization for these DMD treatments.

Policy Summary

PayerPremera Bluecross

PolicyPharmacologic Treatment of Duchenne Muscular Dystrophy

Policy CodePolicy 5.01.570

Change TypeMaterial additions and revisions to coverage criteria and age limits (multiple drugs added/updated)

Effective DateDec. 1, 2025

Next Review DateN/A

Key ActionConfirm genetic mutation amenability and document baseline 6MWT (>=250 m) when requesting prior authorization for exon-skipping drugs or Elevidys.

SourceLink

POLICY UPDATE CHANGES

Added coverage criteria for Elevidys (expanded to age 4 years or older), Agamree (vamorolone) for individuals ≥2 years, Duvyzat (givinostat), and added Pyquvi and Jaythari (deflazacort); policy revised with effective date Dec. 1, 2025 (last revised Nov. 11, 2025).

Updated Elevidys age requirement from a narrow 4–5 year subgroup to indicate Elevidys use for ambulatory pediatric individuals aged 4 years or older and clarified that Elevidys use in non-ambulatory individuals is investigational and not covered.

Added requirement to try generic deflazacort before branded deflazacort products (Emflaza) and added Agamree coverage for individuals ≥2 years; required adequate trials (3 continuous months) for some corticosteroid alternatives.

4Exon-skipping listed

1Gene therapy listed

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≥2506MWT requirement (m)

J1413–J1429HCPCS codes

IncludedAppendix examples

>=3New drug additions since 2023

Coverage Criteria

Site-of-Service and general coverage notes — medical necessity and site-of-service considerations

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Initial Therapy Criteria

Reauthorization and Continuation Criteria

Reauthorization criteria

Reauthorization depends on demonstrated clinical benefit:

Reauthorization for exon-skipping agents: For Amondys 45, Exondys 51, and Vyondys 53: reauthorization up to 12 months if the individual does not show deterioration on two successive 6MWT measurements separated by 6 months over a year compared to baseline OR shows deterioration at a rate less than expected by natural history. For Viltepso: reauthorization up to 12 months based on two successive time-to-stand measurements at 6-month intervals versus baseline. Reauthorization for other agents requires documentation of stable or improved muscle strength relative to baseline.

Continuation therapy notes

Continuation therapy notes and monitoring considerations:

Continuation and safety monitoring: Continuation decisions consider long-term safety and adverse reaction profiles observed in extension studies; for Exondys 51, common adverse events (>=10%) included vomiting, contusion, excoriation, arthralgia, rash, catheter site pain, and URTI, and 50 mg/kg weekly dosing is not recommended. For Elevidys, monitor liver function tests, platelets, and for signs of immune-mediated myositis or myocarditis; corticosteroid management around infusion per trial protocol. Documentation of continued clinical response is required for reauthorization of other agents.

Coding

Covered HCPCS CodesHCPCSCovered

J1413	Injection, delandistrogene moxeparvovec-rokl, per therapeutic dose (Elevidys)
J1426	Injection, casimersen, (Amondys 45), 10 mg
J1427	Injection, viltolarsen, (Viltepso) 10 mg
J1428	Injection, eteplirsen (Exondys 51), 10 mg
J1429	Injection, golodirsen (Vyondys 53), 10 mg

Referenced HCPCS/HCPC codes from policy historyHCPCS

J1428	HCPCS code added 01/01/18 (as noted in history)
J1429	HCPCS code added 07/01/20 (as noted in history)
J1413	HCPCS code added 01/01/24 (as noted in history)
J3590	HCPCS code referenced/added/removed in various history entries
C9070	HCPCS/C code referenced in history
C9075	HCPCS/C code added 07/01/21 (as noted in history)
C9399	HCPCS code added 10/01/23 (as noted in history)
J1426	HCPCS code with term date referenced in history
J1427	HCPCS code added per 04/01/21 history

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

Threshold6-minute walk distance (6MWT) >= 250 meters required for initial authorization (record of baseline 6MWT required)

Applies toAmondys 45, Exondys 51, Vyondys 53, and Elevidys initial therapy criteria

DocumentationBaseline 6MWT result must be included in medical records submitted for review

Ambulation requirementIndividual must be able to ambulate (with or without assistance; assistance must not include wheelchair) to meet 6MWT requirement

6MWT Baseline Example — Study 1 Cohort

Study exampleStudy 1 cohort mean baseline 6-minute walk distance (6MWD) = 363 meters

Provider Actions & Prior Authorization

Prior Authorization

Prior Authorization Durations & Reauthorization Basis

Prior authorization is required for the drugs listed in this policy. Initial approval durations: non-formulary exception reviews may be approved up to 12 months. Initial approvals for Amondys 45 (casimersen), Exondys 51 (eteplirsen), Viltepso (vitolarsen), and Vyondys 53 (golodirsen) may be approved up to 12 months. Initial approvals for Agamree (vamorolone), Duvyzat (givinostat), Emflaza (deflazacort), Jaythari (deflazacort), and Pyquvi (deflazacort) may be approved up to 6 months. Elevidys (delandistrogene moxeparvovec-rokl) may be approved as a one-time infusion. Reauthorization depends on documented clinical benefit/response at time of reauthorization (see items). Repeat treatment with Elevidys is considered investigational except for one-time infusion approvals noted.

Non-formulary exception reviews for all listed drugs may be approved up to 12 months.
Amondys 45, Exondys 51, Viltepso, Vyondys 53: approvals up to 12 months.
Agamree, Duvyzat, Emflaza, Jaythari, Pyquvi: approvals up to 6 months for initial reviews; reauthorization up to 12 months with documentation of continued clinical response.
Elevidys: one-time infusion approval; repeat treatment is investigational (re-authorization limited to non-formulary exception reviews up to 12 months).

Step Therapy

Step	Requirement
1	Adequate trial defined as 3 continuous months required before coverage of some non-preferred corticosteroids; must fail prednisone and generic deflazacort (trial failure defined as lack of response or increase in adverse events).

Recommendation	Rationale / When to Consider
Consider alternative therapies and enrollment in clinical trials where appropriate	Evidence is inconclusive for some exon‑skipping agents and pooled analyses are inconclusive; consider clinical trial enrollment or alternative therapies when demonstrated clinical benefit is limited or uncertain.

Step	Requirement
1	Try generic deflazacort first prior to coverage of branded Emflaza or other branded deflazacort formulations (policy updated to require trial of generic deflazacort).

Site of Care

Billing Rule

Use office, infusion center, or home infusion as preferred sites

Preferred sites of administration are physician office, infusion center, or home infusion when safe and appropriate; hospital outpatient is reserved for initial courses, re‑initiations after ≥6 months, or when outpatient/home options are unavailable within 50 miles or clinical risk factors exist.

Note

Infusion center site‑of‑service review limited to ages ≥13

Site‑of‑service review for infusion center care is limited to individuals aged 13 years and older; pediatric infusion training and requirements differ for younger children.

Documentation Required

Document Elevidys single infusion and peri‑infusion corticosteroid escalation

Definitions

6MWT / 6MWD — Definition

Definition6-Minute walk distance (6MWD): distance (in meters) walked in 6 minutes; used to estimate disease burden and baseline/functional status

PurposeUsed as a clinical outcome measure to assess ambulation and functional capacity in DMD

Use in policyBaseline and follow-up 6MWT results required for Amondys 45, Exondys 51, Vyondys 53 and Elevidys documentation

Antisense Oligonucleotide (AON) — Definition & Role

DefinitionAntisense oligonucleotide (AON): synthetic polymers that bind to mRNA to alter protein synthesis; used to induce exon skipping in DMD

MechanismAONs hybridize to pre-mRNA sequences to modulate splicing and enable exclusion of mutated exons, producing a truncated but partially functional dystrophin

Background

Duchenne muscular dystrophy (DMD) is an X‑linked genetic disorder caused by mutations in the dystrophin gene that disrupt the reading frame and result in absent or severely reduced dystrophin protein. The condition typically presents in early childhood with progressive skeletal muscle weakness, loss of ambulation over time, and eventual cardiopulmonary compromise. Standard management includes corticosteroids and multidisciplinary supportive care; disease‑modifying pharmacologic approaches covered by this policy include exon‑skipping antisense oligonucleotides and an AAV‑based microdystrophin gene therapy targeted to ambulatory pediatric patients.

Key Metrics & Evidence Highlights

4Exon-skipping drugs listed

1Gene therapy product listed (Elevidys)

2506MWT minimum for initial authorization (meters)

J1413-J1429Covered HCPCS codes summary

Revision History

Policy version updated and effective Dec. 1, 2025. Last revised on Nov. 11, 2025. This header reflects the current policy revision and effective date.

Policy Summary

PayerPremera Bluecross

PolicyPharmacologic Treatment of Duchenne Muscular Dystrophy

Policy CodePolicy 5.01.570

Change TypeMaterial additions and revisions to coverage criteria and age limits (multiple drugs added/updated)

Effective DateDec. 1, 2025

Next Review DateN/A

Key ActionConfirm genetic mutation amenability and document baseline 6MWT (>=250 m) when requesting prior authorization for exon-skipping drugs or Elevidys.

SourceLink

On This Page

PopulationIndividuals in Study 1 had mean age 9.4 years and were on stable corticosteroids for ≥6 months

ContextStudy 1 assessed dystrophin production and included 6MWT as a clinical outcome measure

Documentation Required

Genetic Confirmation & Age Verification

Prior authorization must include documentation confirming the diagnosis of Duchenne muscular dystrophy (DMD) and the specific DMD gene mutation. For exon-skipping therapies, genetic testing must demonstrate an amenable mutation (see appendix examples). Age verification per product-specific criteria is required (for example, Elevidys: aged 4 years or older and ambulatory; Agamree and deflazacort products: aged 2 years or older as applicable).

Genetic testing results documenting the specific DMD gene mutation and amenability to the requested exon-skipping therapy (refer to appendix examples).
Document patient age to confirm product-specific eligibility (e.g., Elevidys ≥4 years; Agamree/deflazacort ≥2 years where applicable).

Prior Authorization

Prior Authorization for Listed DMD Therapies

Prior authorization is required for the DMD therapies listed in this policy and will be reviewed against the policy's clinical criteria and FDA dosing/administration. The medications are subject to the product's FDA dosage and administration prescribing information.

Covered/managed products include: Amondys 45 (casimersen), Exondys 51 (eteplirsen), Viltepso (vitolarsen), Vyondys 53 (golodirsen), Agamree (vamorolone), Duvyzat (givinostat), Emflaza/Jaythari/Pyquvi (deflazacort formulations), Elevidys (delandistrogene moxeparvovec-rokl).
Authorizations will follow each product's FDA dosing and administration information; deviations may require additional justification.

Prior Authorization

Prior Authorization Required for Listed DMD Drugs

Prior authorization is required for the specific DMD drugs named in the policy; approvals and exceptions follow the durations above. Non-formulary exception reviews may be approved up to 12 months. For continuation/re-authorization, clinical measures demonstrating benefit or stability are required.

Non-formulary exception reviews for all drugs listed may be approved up to 12 months.
Re-authorization decisions are based on documented clinical benefit/response (see reauthorization criteria).

Denial Risk

Elevidys Investigational Use

Use of Elevidys in non-ambulatory individuals is investigational and excluded from coverage. Elevidys approvals are limited to a one-time infusion for eligible ambulatory pediatric individuals meeting the policy criteria.

Elevidys may be considered medically necessary only for ambulatory male individuals meeting the policy age and mutation criteria.
Non-ambulatory use and repeat dosing of Elevidys are considered investigational and not covered.

Billing Rule

Site-of-Service Medical Necessity Criteria

Site-of-Service (SOS) Medical Necessity criteria apply to medical benefit reviews for IV and injectable drugs; SOS review applies for individuals aged 13 years and older. SOS criteria do NOT apply to Alaska fully-insured members (see Alaska HB 226 exception). SOS review may be applied to injection drugs as noted. Exceptions for hospital-based outpatient initiation (first 90 days) and CRS patients are described in policy.

SOS Medical Necessity applies to IV/ injectable drug administration sites (physician office, infusion center, home infusion, hospital outpatient) for medical-benefit reviews.
For ages ≥13, site-of-service review will be performed; pediatric individuals <13 are generally excluded from SOS review due to special considerations.
SOS criteria do not apply to Alaska fully-insured members per Alaska HB 226.
Hospital-based outpatient administration may be considered medically necessary for the initial 90 days for initial course or re-initiation after ≥6 months; an exception to SOS requirements exists for certain individuals with cytokine release syndrome (CRS).

Documentation Required

Required Documentation for Medical Necessity Review

Submit complete medical records to support medical necessity reviews. Required documentation includes office visit notes with diagnosis and relevant history, genetic testing results, physical evaluation, medication history, and baseline/ follow-up functional measures as applicable.

Office visit notes documenting diagnosis, relevant history, genetic testing, physical exam, and medication history.
Genetic testing report confirming DMD gene mutation and mutation details (exon deletions/amenability).
Baseline and follow-up functional assessments: 6-minute walk test (6MWT) results for Amondys 45, Exondys 51, Vyondys 53 and Elevidys; time-to-stand measurements for Viltepso; North Star/NSAA or other objective measures as requested.
Documentation of corticosteroid use history and stability where required (see corticosteroid trial requirements).

Documentation Required

Required Clinical Documentation

Clinical documentation must include genetic confirmation, baseline functional measures, and monitored follow-up measures used for reauthorization decisions. For exon-skipping drugs, provide mutation amenability details; for gene therapy and other agents, include baseline and follow-up NSAA/6MWT/time-to-stand as applicable.

Documented baseline 6MWT for applicable drugs and subsequent 6MWT results for reauthorization comparisons.
Time-to-stand measurements for Viltepso where applicable.
NSAA or other functional scores for Elevidys and other therapies as requested.
Include genetic report specifying the exact DMD gene mutation and whether it is amenable to the requested therapy (appendix examples may be used as guidance).

Documentation Required

Genetic Mutation Documentation

Genetic testing results must state the confirmed DMD gene mutation (including exon(s) deleted or mutated) and whether the mutation is amenable to the requested exon-skipping therapy. Use the appendix examples to support amenability determinations.

Include the genetic test report with specific exon deletion or mutation details.
When requesting exon-skipping therapies, identify which exon skipping is intended (e.g., exon 45, 51, 53) and how the member's mutation maps to amenable deletions (see appendix).

Step Therapy

Required Trials Before Coverage of Certain Corticosteroids

Before coverage of certain corticosteroids (Agamree, Emflaza, Jaythari, Pyquvi, generic deflazacort), members must have had adequate trials of prednisone and generic deflazacort with documented treatment failure or intolerance. An adequate trial is defined as 3 continuous months of therapy.

Adequate trial = 3 continuous months of therapy.
Require documentation of treatment failure (lack of response or increased adverse events) with prednisone and with generic deflazacort prior to approving branded deflazacort products or Agamree where applicable.
Generic deflazacort is preferred and must be tried prior to Emflaza/Agamree per updated policy. Exceptions should be justified in documentation.

Documentation Required

Evidence-Based Authorization Consideration

Evidence limitations for some therapies (for example, eteplirsen/Exondys 51) may require additional rationale and supporting documentation for authorization. Approvals and reauthorizations will consider the available clinical evidence and documented patient response on objective measures.

Eteplirsen (Exondys 51): clinical benefit has not been established in trials; provide clinical rationale and objective measures (6MWT, other functional data) to support continued use.
Re-authorization decisions for all therapies will consider documented stability or improvement versus expected natural history (e.g., no deterioration on two successive measures 6 months apart over a year, or decline at slower-than-expected rate).

Step Therapy

Generic Deflazacort Preferred

Generic deflazacort (oral tablet and suspension) is preferred and must be tried prior to branded deflazacort products (Emflaza, Agamree exception rules apply) per the updated policy. Documentation of trial and failure/intolerance of generic deflazacort is required when requesting branded formulations.

Require documented adequate trial and treatment failure or intolerance of generic deflazacort before Emflaza, Jaythari, or Pyquvi are approved.
Policy applies to both oral tablet and oral suspension formulations.

Note

Provider/Policy History Notes (Site-of-Service & Other Changes)

Policy history and administrative notes: site-of-service review additions, age updates for Elevidys, product coverage additions (Agamree, Duvyzat, generic deflazacort, Pyquvi, Jaythari), and SOS Alaska exception (HB 226) are noted. Providers should review the policy history for effective dates and applicability.

Site-of-service reviews added for certain drugs with specific effective dates (see history).
Age requirement for Elevidys updated to 4 years or older.
Site-of-service Medical Necessity criteria does not apply to Alaska fully-insured members per Alaska HB 226 as noted in the 2025 update.

For Elevidys administration, note it is a single IV infusion with trial‑specified peri‑infusion corticosteroid escalation (baseline dose increased to at least 1 mg/kg/day starting one day prior and continued for at least 60 days after infusion); include this corticosteroid plan in documentation.

Note

Confirm state‑level exceptions and site‑of‑service variability

Site‑of‑service rules and exceptions may vary by state and specific circumstances; confirm applicable state exceptions and employer/member contract provisions when requesting authorization.

Note

Site‑of‑Service criteria apply to infusion—and from Oct 3, 2025—certain injection drugs

Site‑of‑Service Medical Necessity criteria apply to infusion drugs and, effective Oct 3, 2025, can also apply to injection drugs; note the Alaska exception and that these criteria will be used in medical necessity reviews where applicable.

Subclass examplePhosphorodiamidate morpholino oligomers (PMOs) are a subclass of AON chemistry used for exon-skipping therapies (e.g., eteplirsen, viltolarsen, golodirsen)

Dystrophin Gene / DMD Genetics

GeneDystrophin gene (DMD): very large gene with 79 exons; mutations (commonly deletions of exons 45–55) disrupt the reading frame and cause absent dystrophin in DMD

Pathogenic mechanismFrameshifting exon deletions lead to loss of functional dystrophin and progressive muscle degeneration

TestingGenetic testing is required to confirm the specific DMD gene mutation and determine amenability to exon skipping

PMO — Phosphorodiamidate Morpholino Oligomer

ChemistryPhosphorodiamidate morpholino oligomer (PMO): a charge-neutral antisense oligonucleotide chemistry designed to bind pre-mRNA and induce exon skipping

PropertiesPMOs have minimal protein interaction, resist nuclease degradation better than some AOs, and do not require catalytic proteins for activity

ExamplesPMO subclass includes eteplirsen and other exon-skipping therapeutics referenced in this policy

Elevidys — Product Description

ProductElevidys (delandistrogene moxeparvovec-rokl): AAVrh74-based recombinant microdystrophin gene therapy administered as a single IV infusion for ambulatory pediatric individuals with DMD

VectorRecombinant, non-replicating AAV serotype rh74 capsid delivering a microdystrophin expression cassette

Indication contextEvaluated in pediatric male individuals aged 4–7 years in trials; baseline corticosteroid escalation used peri-infusion in studies

AAV-Based Recombinant Gene Therapy — delandistrogene moxeparvovec-rokl

DescriptionAAV-based recombinant gene therapy delivering a microdystrophin transgene (delandistrogene moxeparvovec-rokl)

AdministrationSingle IV infusion via peripheral limb as used in Elevidys clinical studies

Trial considerationsPeri-infusion corticosteroid escalation (to ≥1 mg/kg daily) was used starting one day prior and continued ≥60 days post-infusion in study protocol

Agamree (vamorolone) — Agent Description

AgentAgamree (vamorolone): a dissociative corticosteroidal anti-inflammatory drug designed to retain anti-inflammatory (transrepression) effects while reducing transactivation-related adverse effects

Mechanism rationaleStructural differences (absence of 11β-hydroxyl/carbonyl moiety) alter receptor interactions to potentially reduce corticosteroid-associated adverse effects

Indication noteApproved for treatment of DMD in individuals ≥2 years per policy updates and supported by VISION-DMD trial data

DMD Exon Deletions Amenable to Exon Skipping — Appendix Examples

DefinitionExon deletions amenable to exon skipping: specific exon deletions in the DMD gene that can be targeted to restore the reading frame (examples provided in appendix)

Examples — exon 45 skippingDeletion of exon 44; deletion of exon 46-47; deletion of exon 46-48; deletion of exon 46-49; deletion of exon 46-51; deletion of exon 46-53; deletion of exon 46-55

Examples — exon 51 & 53 skippingExon 51 examples: deletion of exon 50, 52, 45-50, 47-50, 48-50, 49-50; Exon 53 examples: deletion of exon 52, 45-52, 47-52, 48-52, 49-52, 50-52