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Premera Bluecross DMD Coverage Update & Prior Auth | OpenPayer

ModifiedPremera BluecrossPolicy N/A

Pharmacologic Treatment of Duchenne Muscular Dystrophy

Medical and pharmacy benefit coverage, site-of-service review, and medical necessity criteria for pharmacologic treatments for Duchenne muscular dystrophy (including exon-skipping antisense oligonucleotides, gene therapy, corticosteroids, and other agents) for members of Premera Blue Cross.

Policy Summary

PayerPremera Bluecross

PolicyPharmacologic Treatment of Duchenne Muscular Dystrophy

Policy CodePolicy N/A

Change TypeMaterial coverage updates and age clarifications (added Agamree, Duvyzat, Elevidys age update)

Effective DateOct 3, 2025

Next Review DateN/A

Key ActionSubmit genetic testing and baseline functional measures (6MWT or time-to-stand) with documentation of stable corticosteroid dosing to support prior authorization.

SourceLink

POLICY UPDATE CHANGES

Added coverage criteria for Elevidys for ambulatory pediatric individuals aged 4 through 5 years with DMD; age requirement updated to 4 years or older and clarified Elevidys is investigational in non‑ambulatory individuals.

Added coverage criteria for Agamree (vamorolone) for treatment of DMD in individuals 2 years and older and required trial of generic deflazacort first.

Added coverage criteria for Duvyzat (givinostat) for treatment of certain individuals with DMD.

Clarified site-of-service medical necessity criteria do not apply to Alaska fully‑insured members and updated site‑of‑service clarifications, including injection drugs and CRS exceptions.

J1413,J1426-J1429HCPCS codes listed for covered agents

>=250 mAmbulation threshold

12 moTypical approval duration

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Coverage Criteria and Drug-specific Rules

Amondys 45 (casimersen) initial therapy

Covered when ALL of the following are met:

Amondys 45 initial criteria: The individual is aged 21 years or younger; assigned male at birth; has a diagnosis of Duchenne muscular dystrophy (DMD); has a confirmed DMD gene mutation amenable to exon 45 skipping (genetic testing required); can ambulate (with or without assistance) and complete a baseline 6-minute walk test (6MWT) of at least 250 meters (baseline 6MWT record required); and has been established on a stable dose of corticosteroids for at least 3 months.

Exondys 51 (eteplirsen) initial therapy

Covered when ALL of the following are met:

Exondys 51 initial criteria: The individual is aged 19 years or younger; assigned male at birth; has a diagnosis of Duchenne muscular dystrophy (DMD); has a confirmed DMD gene mutation amenable to exon 51 skipping (genetic testing required); can ambulate (with or without assistance) and complete a baseline 6-minute walk test (6MWT) of at least 250 meters (baseline 6MWT record required); has been established on a stable dose of corticosteroids for at least 3 months; and dosing is limited to 30 mg/kg IV once weekly per policy dosing statement.

Viltepso (viltolarsen) initial therapy

Covered when ALL of the following are met:

Viltepso initial criteria: The individual is aged 10 years or younger; assigned male at birth; has a diagnosis of Duchenne muscular dystrophy (DMD); has a confirmed DMD gene mutation amenable to exon 53 skipping (genetic testing required); can ambulate (with or without assistance) and has a time-to-stand of less than 10 seconds; and has been established on a stable dose of corticosteroids for at least 3 months.

Vyondys 53 (golodirsen) initial therapy

Covered when ALL of the following are met:

Vyondys 53 initial criteria: The individual is 15 years or younger; assigned male at birth; has a diagnosis of Duchenne muscular dystrophy (DMD); has a confirmed DMD gene mutation amenable to exon 53 skipping (genetic testing required); can ambulate (with or without assistance) and complete a baseline 6-minute walk test (6MWT) of at least 250 meters (baseline 6MWT record required); has been established on a stable dose of corticosteroids for at least 3 months; and dose is limited per policy dosing statement (document contains inconsistent dosing passages; policy dosing statements should be followed).

Elevidys (gene therapy) initial therapy

Covered when ALL of the following are met:

Elevidys initial criteria: Male individuals aged 4 years or older; diagnosis of Duchenne muscular dystrophy (DMD); confirmed DMD gene mutation (genetic testing required) without a mutation in exons 1–17; not ventilator dependent; no active heart failure (ejection fraction >= 40%); can ambulate without a wheelchair and complete a baseline 6-minute walk test (6MWT) of at least 250 meters (baseline 6MWT record required); has been established on a stable dose of corticosteroids for at least 3 months prior and maintained for at least 30 days after infusion; and has an anti-AAVrh74 total binding antibody titer less than 1:400.

Corticosteroids and alternatives

Covered when ALL of the following are met:

Corticosteroids and related agents: Agamree (vamorolone) and Emflaza (deflazacort) may be considered medically necessary for individuals aged 2 years or older with DMD. Generic deflazacort may be considered medically necessary for individuals aged 2 years or older with DMD after an adequate trial and treatment failure with prednisone (adequate trial defined as 3 continuous months). Policy changes reduced the corticosteroid stabilization requirement historically from 6 months to 3 months.

Duvyzat (givinostat) initial therapy

Covered when ALL of the following are met:

Duvyzat initial criteria: The individual is aged 6 years or older; has a diagnosis of Duchenne muscular dystrophy (DMD); has a confirmed DMD gene mutation (genetic testing required); has been established on a stable dose of corticosteroids (policy notes a 6-month stabilization in the Duvyzat section); and can ambulate without a wheelchair and complete a baseline 6-minute walk test (6MWT) of at least 250 meters.

Drug-specific coverage updates

Policy updates added or revised coverage criteria for the following agents:

Elevidys coverage update: Added coverage criteria for Elevidys for ambulatory pediatric individuals; initial 2023 language specified ages 4 through 5 years and was updated in 2024 to indicate age 4 years or older; use in non-ambulatory individuals is considered investigational.age >=4 years and ambulatory

2023–2024 updates documented in policy history.

Agamree (vamorolone) and deflazacort update: Added coverage criteria for Agamree for individuals 2 years of age and older; updated Agamree and Emflaza (deflazacort) criteria to require trial of generic deflazacort first; added generic deflazacort coverage with requirement of prior prednisone trial (adequate trial = 3 months).age >=2 years

2024 update.

Duvyzat coverage: Added coverage criteria for Duvyzat (givinostat) for certain individuals with DMD; pivotal trial enrollment and safety monitoring referenced in prescribing information.

All uses of the medications listed in this policy that are not explicitly described in the coverage criteria are considered investigational. This includes, but is not limited to, use of Amondys 45 (casimersen), Exondys 51 (eteplirsen), Viltepso (viltolarsen), and Vyondys 53 (golodirsen) for indications outside those specified here, and use of those exon‑skipping agents after Elevidys infusion.

The policy states that the clinical benefit of Exondys 51 (eteplirsen) has not been established in clinical trials; therefore use of Exondys 51 outside the narrowly defined, drug‑specific coverage criteria should be considered investigational or of uncertain medical necessity.

Use of Elevidys (delandistrogene moxeparvovec‑rokl) in individuals who are non‑ambulatory is explicitly considered investigational and is not covered under the policy.

This medical policy does not apply to Medicare Advantage. Providers and members should consult member benefit documents or contact customer service to determine coverage under Medicare Advantage plans and any plan‑specific rules.

Site‑of‑service reviews apply to IV and injectable DMD therapies; infusion or injection services provided at a site that does not meet the policy's site‑of‑service criteria are considered not medically necessary. Preferred medically necessary sites include physician office, infusion center, home infusion, and hospital outpatient infusion departments, with hospital outpatient care reserved for initial courses, re‑initiations after ≥6 months, or when outpatient/home infusion is unavailable or the patient has clinical conditions that increase infusion risk.

The document reiterates that evidence supporting Exondys 51 is limited: the clinical benefit of Exondys 51 (eteplirsen) has not been established in trials, which underpins the policy’s cautious coverage stance and investigational classification for uses outside specified criteria.

The policy explicitly states that Elevidys is investigational for non‑ambulatory individuals. Coverage criteria and updates clarify Elevidys is intended for ambulatory pediatric individuals meeting the policy’s age and functional requirements; treatment of non‑ambulatory patients is not supported by this policy.

Initial Authorization Requirements

Initial therapy requirements

Initial authorization requires meeting ALL of the following common elements (drug-specific age, genetic, functional, and corticosteroid stabilization criteria):

Initial therapy common elements: Genetic confirmation of DMD with a mutation amenable to the specific exon skipping or gene therapy product (genetic testing required); male individuals within drug-specific age limits; baseline functional measure as specified by the drug (6MWT >=250 meters for most exon‑skipping agents and Elevidys; time-to-stand <10 seconds for Viltepso when specified); and establishment on a stable corticosteroid regimen for the duration required by the specific drug (policy-wide stabilization requirement is at least 3 months unless otherwise specified).

Drug-specific functional and age thresholds: Follow the drug-specific age and functional thresholds in each drug section (examples: Amondys 45 age ≤21 and 6MWT >=250 m; Exondys 51 age ≤19 and 6MWT >=250 m; Viltepso age ≤10 and time-to-stand <10 s; Vyondys 53 age ≤15 and 6MWT >=250 m; Elevidys age >=4 years and 6MWT >=250 m).

Corticosteroid stabilization note: Policy history reduced the corticosteroid stabilization requirement from 6 months to 3 months; follow the current requirement of stable corticosteroid dose >=3 months unless a drug-specific longer duration is specified in the drug section.

Reauthorization and Ongoing Treatment Criteria

Reauthorization criteria

Reauthorization may be approved when the following are met (agent-dependent):

Reauthorization for exon-skipping agents: Reauthorization for Amondys 45, Exondys 51, Vyondys 53, and Viltepso may be approved up to 12 months and depends on demonstrated clinical benefit/response: the individual does not show deterioration on two successive functional measures (6MWT for most agents or time‑to‑stand for Viltepso) spaced approximately 6 months apart over a year compared to baseline, OR shows deterioration at a rate less than expected based on natural history.

Reauthorization for corticosteroids and other oral agents: Non-formulary exception reviews and reauthorizations for Agamree (vamorolone), Duvyzat (givinostat), and Emflaza (deflazacort) may be approved up to 12 months and require documentation of continued clinical response measured by improvement or stable muscle strength compared to baseline and/or diminished loss of muscle strength.

Elevidys continuation

Elevidys continuation and long-term considerations:

Step Therapy Preferences and Historical Requirements

Requirement	Details
Prednisone trial required before generic deflazacort
Generic deflazacort may be considered medically necessary only after an adequate trial and treatment failure with prednisone (adequate trial defined as 3 continuous months).

Statement	Notes
Glucocorticoids are standard supportive therapy for Duchenne muscular dystrophy (DMD)
Long-term glucocorticoid use has been shown to delay loss of ambulation, preserve upper limb and respiratory function, and reduce scoliosis risk; however, no formal step therapy sequence is specified in the policy.

Preference	Policy detail
Try generic deflazacort first
2024 update added a requirement to try generic deflazacort before Emflaza (deflazacort) or Agamree (vamorolone) where specified; generic deflazacort coverage criteria require age ≥2 years and prior prednisone trial per policy.

Historical change	Effect on corticosteroid stabilization requirement
Stabilization duration reduced from 6 to 3 months
Policy history documents that the required duration an individual be established on a stable corticosteroid dose was reduced from 6 months to 3 months (noted in P&T/criteria updates).

Billing and 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.

HCPCS/J-code history for DMD drugsmixed

J1428	HCPCS code J1428 referenced (added 01/01/18)
J1429	HCPCS code J1429 (added 07/01/20)
J1413	HCPCS code J1413 (added 01/01/24)
J1426	HCPCS code J1426 (added 11/01/21; effective date removed later)
J1427	HCPCS code J1427 (added 04/01/21)
J3590	HCPCS J3590 referenced (added/removed at various times for different products)
C9070	HCPCS C9070 (term date added 04/01/21)
C9071	HCPCS C9071 (added 01/01/21; later removed 09/01/22)
C9075	HCPCS C9075 (added 07/01/21; later removed)
C9399	HCPCS C9399 (added for Elevidys 10/01/23; later removed)

inv-18: 6-Minute Walk Test (6MWT) threshold used in multiple therapies

Threshold6-minute walk distance (6MWT/6MWD) >= 250 meters

PurposeUsed as a baseline functional measure to document ambulatory status for initial authorization of multiple DMD therapies (Amondys 45, Exondys 51, Vyondys 53, Elevidys, Duvyzat).

DocumentationRecord of the baseline 6MWT is required for initial review and authorization.

Use in reauthorizationReauthorization for exon-skipping agents may require serial 6MWT measures to demonstrate absence of deterioration on two successive measures spaced 6 months apart or a rate of decline less than natural history expectations.

inv-19: Viltepso time-to-stand threshold

Threshold

Prior Authorization, Documentation, and Provider Steps

Prior Authorization

Prior Authorization and Site‑of‑Service

Prior authorization is required for the drugs listed in this policy. Authorizations may be issued for initial and reauthorization reviews; non‑formulary exception reviews and many authorizations can be approved up to 12 months (some agents/devices have shorter default approval intervals per policy history). Site‑of‑service medical necessity review applies to IV and injectable drugs for medical‑benefit reviews and may result in denial if the service location does not meet the SOS criteria. Verify member benefits and plan limits prior to submission — benefit exclusions or limits may trigger a denial even when medical necessity criteria are met.

Initial and reauthorization reviews may be approved up to 12 months for many agents; some drugs (e.g., Agamree, Duvyzat, Emflaza historically) have shorter default review durations (see policy history).
Site‑of‑service (SOS) medical necessity review applies to IV/injectable administration and can be denied if SOS criteria are not met; SOS review does NOT apply to Alaska fully‑insured members per Alaska HB 226.
Non‑formulary exception reviews for all drugs listed may be approved up to 12 months.
An SOS exception exists for certain individuals receiving treatment for cytokine release syndrome (CRS).

Site-of-Service Rules and Exceptions

Note

Select medically necessary site: office, infusion center, hospital outpatient, or home

Preferred medically necessary sites include physician office, infusion center, home infusion, and hospital outpatient; site‑of‑service medical necessity review applies to IV and injectable agents and should be considered when planning treatment location.

Hospital outpatient is considered medically necessary for initial courses, re‑initiation after ≥6 months, lack of local outpatient/home infusion, or significant clinical risk factors.
Home infusion and infusion centers are listed as preferred medically necessary sites when policy criteria are met.

Note

Infusion center reviews apply for individuals ≥13 yrs; Elevidys is single IV infusion in clinical setting

Site‑of‑service review is limited to individuals aged 13 years or older; Elevidys is administered as a single IV infusion in a clinical setting (per trial) and site‑of‑service rules apply where age criteria are met.

Elevidys was administered as a single peripheral limb IV infusion in trials; document infusion setting and corticosteroid stabilization per protocol when applicable.

Definitions and Background Terms

Duchenne muscular dystrophy (DMD) is an X‑linked genetic disorder caused by mutations in the DMD gene leading to absence or deficiency of functional dystrophin. Loss of dystrophin destabilizes the muscle cell membrane, causing progressive muscle degeneration and weakness that typically begins in early childhood and later involves respiratory and cardiac muscles. Clinical assessment commonly uses genetic testing to confirm DMD and functional measures such as the 6‑minute walk test (6MWT) and other motor scales to document ambulatory status and disease progression.

inv-37: 6-Minute walk distance test (6MWD) definition

Term6-Minute walk distance test (6MWD)

DefinitionTest that measures the distance (in meters) a person can walk in 6 minutes.

Clinical useHelps estimate disease burden and ambulatory function; used as a baseline and serial measure in DMD drug authorization and trial endpoints.

inv-38: Antisense oligonucleotide (AON) definition

Policy Revision History

2017-04-01policy_created/annual_review

Policy created / annual review with P&T Committee updates and initial site-of-service and coding tracking beginning in 2017.

2018-01-01coding_update

Added HCPCS code J1428 (eteplirsen) to the policy coding list effective 2018-01-01.

2018-02-01site_of_service_update

Exondys 51 (eteplirsen) became subject to site-of-service review for administration as of mid-2018 per interim review.