CurrentPremera BluecrossPolicy 5.01.570

5.01.570 Pharmacologic Treatment of Duchenne Muscular Dystrophy

Defines medical-necessity/site-of-service criteria, coverage, prior authorization durations, documentation and coding for specified DMD pharmacologic therapies (antisense oligonucleotides, gene therapy, corticosteroids, histone deacetylase inhibitor) and distinguishes medical vs pharmacy benefit management.

Policy Summary

PayerPremera Bluecross

Policy5.01.570 Pharmacologic Treatment of Duchenne Muscular Dystrophy

Policy CodePolicy 5.01.570

Change TypeAuthorization, criteria, quantity limits

Effective DateMay 1, 2026

Next Review Date

Key ActionSubmit office visit notes with diagnosis, genetic testing, medication history and baseline/ongoing functional measures (6MWT, time to stand) as applicable.

SourceLink

POLICY UPDATE CHANGES

Updated initial authorization duration for Agamree (vamorolone), Duvyzat (givinostat), Emflaza (deflazacort), Jaythari (deflazacort), and Pyquvi (deflazacort) from 6 months to 12 months.

Added quantity limit for Agamree (vamorolone) of 9 grams (225 ml) per 30 days.

Added coverage criteria for Pyquvi (deflazacort) and Jaythari (deflazacort).

Updated re-authorization coverage criteria to require documentation that the individual is ambulatory without needing an assistive device for several drugs (Amondys 45, Duvyzat, Exondys 51, Viltepso, Vyondys 53).

Updated Elevidys age requirement from '4 through 5 years' to '4 years or older' and clarified use in non-ambulatory individuals is investigational.

Clarified that medications listed in this policy are subject to the product's FDA dosage and administration prescribing information.

Site of Service Medical Necessity criteria does not apply to Alaska fully-insured members pursuant to Alaska HB 226.

Updated initial steroid-stable-duration requirement from 6 months to 3 months (historical change in 2020); later updates adjusted authorization durations—current specific durations reflected in Part 1 criteria.

Added appendix with examples of DMD gene mutations amenable to exon 45, 51, and 53 skipping.

5Drugs with medical-benefit management listed

6Drugs managed through pharmacy benefit listed

250Common 6MWT ambulatory threshold (meters)

3Exon skipping categories with appendix examples

12 moUpdated initial authorization duration for select drugs

Coverage Summary

General requirements

Covered when ALL of the following are met:

ALL of the following

Individual has a diagnosis of Duchenne muscular dystrophy (DMD).
Genetic testing confirms a mutation of the DMD gene that is amenable to the specific therapy requested (e.g., exon-target or gene therapy vector as applicable).
Genetic test report must be included in documentation.
Patient is assigned male at birth.
Policy applies to individuals meeting clinical trial/label criteria; exceptions documented in drug-specific nodes below.
Age requirement (drug-specific)
- Elevidys (delandistrogene moxeparvovec-rokl): age and other label criteria per drug-specific group below.
- Antisense oligonucleotides (eteplirsen, golodirsen, casimersen, vitolarsen): age limits per drug-specific group below.
- Corticosteroids or alternatives (prednisone, deflazacort, vamorolone): age ≥2 years for some agents; see corticosteroid group below.
Baseline functional assessments appropriate to the agent requested (e.g., baseline 6-minute walk test [6MWT] or time to stand) when required by the drug-specific criteria.
Record of baseline 6MWT or timed function test is required for initial review when specified by the drug-specific criteria.
Documented trial and/or stable use of corticosteroids when required by the drug-specific criteria (minimum duration where specified).
Dose and administration limits follow the drug-specific criteria (eg, mg/kg and frequency limits).
Site of service review applies for IV/injectable agents per the Site of Service Medical Necessity criteria; hospital-based outpatient setting may be required for initial or high-risk situations.

Site of Service Medical Necessity (general)

ALL of the following

Site of Service (SOS) criteria apply only to medical benefit reviews (not applicable to Alaska fully-insured members for SOS; refer to infusion/injection drug medical necessity criteria for Alaska fully-insured).
SOS review applies to IV and injectable DMD therapies listed in this policy (including Amondys 45, Exondys 51, Vyondys 53, Viltepso, and Elevidys).
Medically necessary sites of service
- Physician's office.
- Infusion center.

Elevidys (delandistrogene moxeparvovec-rokl)

Elevidys (delandistrogene moxeparvovec-rokl) coverage criteria

ALL of the following

Indication and age per FDA label: single intravenous infusion for treatment of ambulatory males with DMD caused by mutations in the DMD gene amenable to dystrophin expression by micro-dystrophin transgene, and within the labeled age range.
Genetic testing confirming mutation amenable to micro-dystrophin gene therapy; report required.
Patient is ambulatory at the time of infusion (as defined by ability to ambulate with or without assistance); baseline functional status documented.
No prior exposure to AAVrh74 or other vector-specific contraindications per labeling; screening for anti-AAVrh74 antibodies per manufacturer guidance and documented.

Corticosteroids and alternatives

Corticosteroids and alternatives (coverage criteria and limits)

ALL of the following

Indication: diagnosed Duchenne muscular dystrophy (DMD).
Agents covered and key requirements
- Prednisone (oral): standard of care; dosing per guideline and prescriber judgment.
- Emflaza (deflazacort) / generic deflazacort: covered when medically indicated; note dosing limits and prescribing information.
- Agamree (vamorolone): may be considered medically necessary if ALL the following are met: individual is aged ≥2 years; diagnosed with DMD; has had an adequate trial and treatment failure due to lack of response or increased adverse events with prednisone; AND has had an adequate trial and treatment failure due to lack of response or increased adverse events with generic deflazacort. Prescribed quantity limited to 9 grams (225 ml) per 30 days.

Duvyzat (givinostat)

Duvyzat (givinostat) coverage considerations

ALL of the following

Duvyzat (givinostat) is considered per evidence and label for use in DMD when applicable; confirm indication and patient meets any trial or label-specified criteria.
Genetic confirmation of DMD required; documentation of baseline functional assessments and labs as required by the product's safety monitoring parameters.
Assess prior and concurrent therapies (including corticosteroids); document rationale for givinostat use (eg, clinical trial eligibility, lack of response/intolerance to standard therapies) when applicable.
Monitor for known toxicities per manufacturer guidance; dosing and administration according to FDA label or evidence-based protocols.

Initial Therapy Criteria (drug-specific)

Amondys 45 (casimersen) - Medical Necessity for treatment/site of service

May be considered medically necessary when ALL are met:

Individual is aged 21 years or younger

Assigned male at birth

Diagnosis of Duchenne muscular dystrophy (DMD)

Confirmed DMD gene mutation amenable to exon 45 skipping; genetic testing required for definitive diagnosis

Refer to appendix for examples

Can ambulate (with or without assistance) and complete a 6-minute walk distance (6MWD) test of at least 250 meters>= 250 meters

Continuation / Re-authorization Criteria

Authorization durations

Initial and re-authorization limits:

Initial authorization: All drugs except Elevidys may be approved up to 12 months12 months

Updated initial durations in 2026 where applicable

Elevidys: approved as a one-time infusionone-time

Repeat treatment considered investigational

Re-authorization ambulatory: Re-authorization: Amondys 45, Duvyzat, Exondys 51, Viltepso, Vyondys 53 may be approved up to 12 months when documentation shows the individual is ambulatory without needing an assistive deviceambulatory without assistive device

Investigational / Not Covered Uses

Listed investigational exclusions:

All uses of Agamree, Duvyzat, Emflaza, Jaythari, Pyquvi beyond policy-described indications are investigational

All uses of Amondys 45, Exondys 51, Viltepso, Vyondys 53 beyond policy-described indications are investigational, including use after Elevidys infusion

All other uses of Elevidys beyond outlined indications are investigational; Elevidys for non-ambulatory individuals is investigational; repeat Elevidys treatment is investigational

Applicable Codes

HCPCS codes for drugs in policyHCPCS

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/Coding referenced historically and for gene therapy/drugsmixed

J1426	HCPCS code for casimersen (Amondys 45) — listed in coding history and policy
J1427	HCPCS code for viltolarsen (Viltepso) — added in coding updates
J1428	HCPCS code for eteplirsen (Exondys 51) — coding update effective 01/01/18
J1413	HCPCS code for delandistrogene moxeparvovec-rokl (Elevidys) — added 01/01/24
J1429	HCPCS code for golodirsen (Vyondys 53) — added 07/01/20
J3590	Intermittently used HCPCS code historically (added/removed for Elevidys/Viltepso in prior updates)
C9070	HCPCS code term date noted in coding history (historical entry)
C9071	HCPCS code added then later removed (historical)
C9075	HCPCS code added then terminated (historical)
C9399	HCPCS code previously added for Elevidys then removed (historical)

Provider Actions / Prior Authorization Requirements

Prior Authorization

Prior Authorization / Site-of-Service Review Required

Site-of-service review and prior authorization required for all IV/injectable DMD therapies and for site-of-service review for individuals aged 13 years and older. Medical necessity review will determine the appropriate administration location (physician office, infusion center, hospital outpatient, or home infusion) and any exceptions. Contact Customer Service for Alaska fully-insured member guidance.

Site-of-service review applies to Amondys 45, Exondys 51, Vyondys 53, Viltepso and other IV/injectable DMD drugs.
For age ≥13 years, site-of-service medical necessity assessment is required.

Documentation Required

Provide Baseline and Ongoing Functional Measures

Provide baseline documentation at initiation and ongoing functional measures for monitoring. Required baseline and ongoing documentation must be submitted with requests.

Clinical Evidence Highlights

0.28%Exondys 51 mean change in dystrophin at Week 48 (baseline 0.16% → 0.44%; p=0.008)

5.3%Viltepso mean dystrophin increase at Week 25 (80 mg/kg weekly; p=0.01)

1.736%Amondys 45 interim ESSENCE Week 48 mean dystrophin increase (from 0.925% baseline; p<0.001)

metAgamree VISION‑DMD primary endpoint TTSTAND velocity met (6 mg/kg/day vs placebo; P=0.002)

Evidence summaries show modest to variable biomarker and expression changes across DMD therapies: Exondys 51 reported a mean change in dystrophin 0.28% (baseline 0.16% to 0.44%), p=0.008; Viltepso (80 mg/kg weekly) produced a mean dystrophin increase 5.3% of normal at Week 25 (p=0.01); Amondys 45 interim ESSENCE 48-week results showed a mean dystrophin increase 1.736% from baseline 0.925% (p<0.001); and Elevidys demonstrated substantially higher micro-dystrophin expression in study cohorts (reported mean changes ~43.4, 40.7, and 54.2 across study parts).

Background

Duchenne muscular dystrophy (DMD) is an X-linked disorder caused by mutations in the DMD gene that commonly lead to absent dystrophin and progressive muscle degeneration affecting skeletal, cardiac, and respiratory muscles. Exon-skipping approaches use antisense oligonucleotides (AONs) — synthetic polymers that bind mRNA — to skip targeted exons and produce a truncated but partially functional dystrophin protein (examples: exon 45, 51, 53 skipping); gene therapy (e.g., AAV-based micro-dystrophin transfer) aims to restore dystrophin expression via viral vectors. Corticosteroids (prednisone, deflazacort) remain standard therapy to slow strength decline and preserve pulmonary/orthopedic function but carry recognized long-term adverse effects (fluid retention, immunosuppression, osteoporosis, impaired glucose metabolism, growth suppression, etc.).

Definitions: 6-Minute Walk Distance (6MWD) — test measuring meters walked in 6 minutes to estimate ambulation/disease burden; Antisense oligonucleotide (AON) — synthetic molecules that alter protein synthesis by binding mRNA; Exon skipping — molecular strategy to skip a specific exon to yield a truncated, partially functional dystrophin (examples: exon 45/51/53 skipping; appendix lists amenable deletions); Ambulatory — able to walk without needing an assistive device (e.g., cane, walker, wheelchair) — documentation of ambulation is required at re-authorization for certain drugs; Exon skipping amenable mutation — a specific DMD exon deletion(s) correctable by skipping the target exon (examples given for exons 45, 51, 53).

Gene Therapy / Safety Considerations

Gene therapy / Safety considerations (Elevidys warnings)

Informational/coverage-limiting safety findings relevant to Elevidys:

FDA safety communication (June 24, 2025) investigating reports of fatal acute liver failure in non-ambulatory pediatric male patients after Elevidys

Led to distribution hold requests and investigations; voluntary hold removed for ambulatory patients July 28, 2025

Severe adverse events reported: acute serious liver injury, immune-mediated myositis, myocarditis, thrombocytopenia, elevated LFTs, vomiting, nausea, pyrexia

Providers should consider these risks when determining medical necessity

Key Thresholds & Quantity Limits

Revision History

2026-05-01effectiveLatest

Updated initial authorization duration for Agamree (vamorolone), Duvyzat (givinostat), Emflaza (deflazacort), Jaythari (deflazacort), and Pyquvi (deflazacort) from 6 months to 12 months; added quantity limit for Agamree (vamorolone) of 9 grams (225 ml) per 30 days (approved April 27, 2026).

2026-04-27approvalLatest

Policy revisions approved updating initial authorization durations and Agamree quantity limit (effective 05/01/26).

2026-03-04

Policy Summary

PayerPremera Bluecross

Policy5.01.570 Pharmacologic Treatment of Duchenne Muscular Dystrophy

Policy CodePolicy 5.01.570

Change TypeAuthorization, criteria, quantity limits

Effective DateMay 1, 2026

Next Review Date

Key ActionSubmit office visit notes with diagnosis, genetic testing, medication history and baseline/ongoing functional measures (6MWT, time to stand) as applicable.

SourceLink

On This Page

Home infusion when a contracted home infusion agency can safely provide the service; otherwise alternate sites per criteria.

Hospital-based outpatient setting (outpatient IV infusion department) - considered medically necessary for the initial 90 days for: the initial course of infusion/injection, or re-initiation after ≥6 months discontinuation (excluding standard dosing schedules with intervals ≥6 months).

When hospital-based outpatient site is appropriate

No outpatient infusion center within 50 miles and no contracted home infusion agency willing/able to travel to the individual's home; or hospital is the only place offering the drug infusion/injection.
Individual has a clinical condition increasing risk of complications for infusion/injection, including any of: known cardiac condition (e.g., symptomatic arrhythmia), significant respiratory disease (e.g., obstructive disease, %FVC ≤40%), unstable renal function, difficult/unstable vascular access, acute mental status changes impacting safety, or history of severe adverse reactions/anaphylaxis to related therapy.
Cytokine release syndrome (CRS) grade 3 or 4 with all the following: temperature ≥38 °C, hypotension requiring ≥1 vasopressors, hypoxia requiring high-flow oxygen or positive pressure; and the individual will be admitted to inpatient setting.

Sites considered not medically necessary when SOS criteria are not met.

Pre-infusion evaluation and baseline labs completed and documented, including hepatic and cardiac assessment as required by labeling.

Single-dose administration with dose per FDA label; documentation of site of service meeting SOS criteria for administration (hospital outpatient may be required for initial infusion).

Exclusion: not medically necessary for individuals with contraindications per product labeling or inadequate antibody screening results.

Documentation of trials and reasons for treatment failure (lack of efficacy or adverse events) for alternatives like vamorolone is required.

Continuation/maintenance: ongoing documentation of benefit and monitoring of adverse effects per standard practice; dose limits per product labeling and policy.

Coverage may be limited or considered investigational for uses not supported by labeling or strong evidence; prior authorization documentation should include supporting rationale and clinical records.

Record of baseline 6MWT necessary for initial review

Established on a stable dose of corticosteroids for at least 3 months

Dose limited to 30 mg/kg IV once weekly

Exondys 51 (eteplirsen) - Medical Necessity for treatment/site of service

May be considered medically necessary when ALL are met:

Individual is aged 19 years or younger

Assigned male at birth

Diagnosis of Duchenne muscular dystrophy (DMD)

Confirmed DMD gene mutation amenable to exon 51 skipping; genetic testing required

Refer to appendix for examples

Can ambulate (with or without assistance) and complete a 6-minute walk distance (6MWD) test of at least 250 meters>= 250 meters

Record of baseline 6MWT necessary for initial review

Established on a stable dose of corticosteroids for at least 3 months

Dose limited to 30 mg/kg IV once weekly

Assistance not to include use of a wheelchair

Viltepso (viltolarsen) - Medical Necessity for treatment/site of service

May be considered medically necessary when ALL are met:

Individual is aged 10 years or younger

Assigned male at birth

Diagnosis of Duchenne muscular dystrophy (DMD)

Confirmed DMD gene mutation amenable to exon 53 skipping; genetic testing required

Refer to appendix for examples

Can ambulate (with or without assistance) with a time to stand of less than 10 seconds< 10 seconds

Assistance not to include use of a wheelchair

Established on a stable dose of corticosteroids for at least 3 months

Dose limited to 80 mg/kg IV once weekly

Vyondys 53 (golodirsen) - Medical Necessity for treatment/site of service

May be considered medically necessary when ALL are met:

Individual is aged 15 years or younger

Assigned male at birth

Diagnosis of Duchenne muscular dystrophy (DMD)

Confirmed DMD gene mutation amenable to exon 53 skipping; genetic testing required

Refer to appendix for examples

Can ambulate (with or without assistance) and complete a 6-minute walk distance (6MWD) test of at least 250 meters>= 250 meters

Record of baseline 6MWT necessary for initial review

Established on a stable dose of corticosteroids for at least 3 months

Dose unspecified in this part (refer to product prescribing information)

Elevidys (delandistrogene moxeparvovec-rokl) - Medical Necessity (gene therapy)

May be considered medically necessary for male individuals when ALL are met:

Individual is aged 4 years or older

2024 update expanded age to 4 years or older

Diagnosis of Duchenne muscular dystrophy (DMD)

Confirmed DMD gene mutation; genetic testing required

Does NOT have a mutation in exon 1-17

Is not on the ventilator

Does not have active heart failure (ejection fraction < 40%)EF >= 40%

Can ambulate without a wheelchair and complete a 6MWD test of at least 250 meters>= 250 meters

Record of baseline 6MWT necessary for initial review

Established on a stable dose of corticosteroids for at least 3 months prior and at least 30 days after gene transfer

Peri-infusional steroid stabilization requirement

Has anti-AAVrh74 total binding antibody titer < 1:400< 1:400

Antibody titer requirement

Corticosteroids and related agents - Medical Necessity

Oral corticosteroids and alternatives may be considered medically necessary under listed conditions:

Agamree (vamorolone): individual aged ≥2 years; diagnosed with DMD; adequate trial (3 continuous months) and treatment failure with prednisone; adequate trial and treatment failure with generic deflazacort; quantity limited to 9 grams (225 ml) per 30 days9 grams per 30 days

Quantity-limit note added in 2026 update

Deflazacort branded: Emflaza (deflazacort), Jaythari (deflazacort), Pyquvi (deflazacort): individual aged ≥2 years; diagnosed with DMD; adequate trial (3 continuous months) and treatment failure with prednisone; adequate trial and treatment failure with generic deflazacort

Coverage added for Jaythari and Pyquvi

Generic deflazacort: individual aged ≥2 years; diagnosed with DMD; adequate trial (3 continuous months) and treatment failure with prednisone

Adequate trial defined as 3 continuous months

Elevidys (delandistrogene moxeparvovec-rokl) - peri-infusion regimen and site-of-service notes

May be considered medically necessary when ALL are met (trial-specific peri-infusion regimen and site-of-service notes):

Peri-infusion steroid regimen (trial): Increase baseline corticosteroid to at least 1 mg/kg daily starting 1 day prior to Elevidys infusion and continue for at least 60 days after infusion (per Study 1 regimen)>= 1 mg/kg/day; >=60 days

Providers should document this regimen as used in pivotal trials

Elevidys site-of-service: Site of Service Medical Necessity criteria may apply; Site of Service criteria does not apply to Alaska fully-insured members per Alaska HB 226; CRS exceptions apply for certain cases

Refer to SOS criteria for specifics

Antibody testing: Has anti-AAVrh74 total binding antibody titer < 1:400 prior to infusion< 1:400

Antibody titer required for Elevidys eligibility

Duvyzat (givinostat) - Medical Necessity

May be considered medically necessary when ALL are met:

Individual is aged 6 years or older

Duvyzat age criterion

Has diagnosis of DMD with a confirmed mutation of the DMD gene

Genetic testing required

Established on a stable dose of corticosteroids for at least 6 months>= 6 months

Duvyzat-specific steroid stabilization duration

Can ambulate without a wheelchair and complete a 6MWD test of at least 250 meters>= 250 meters

Baseline required

Requirement added effective March 4, 2026

Re-authorization steroids: Re-authorization: Agamree, Emflaza, Jaythari, Pyquvi may be approved up to 12 months when documentation shows continued clinical response (improvement or stable muscle strength vs baseline and/or diminished loss of muscle strength)continued clinical response

Initial authorization duration for these agents updated to 12 months in 2026

Exon-skipping therapies (class-level requirements referenced)

Criteria for exon-skipping drugs (examples include Amondys 45, Exondys 51, Viltepso, Vyondys 53):

Individual has a DMD mutation amenable to the specific exon skipping (examples provided in appendix for exon 45, 51, 53)

Appendix lists amenable exon deletions

Documentation at re-authorization that the individual is ambulatory without needing an assistive device (e.g., cane, walker, wheelchair)ambulatory without assistive device

Requirement added effective March 4, 2026

Established on a stable dose of corticosteroids for required duration prior to therapy where applicablestable dose duration varies by product (see Part 1 drug-specific criteria)

Steroid stabilization requirement; varied historical edits

Corticosteroid re-authorization (continued clinical response)

Re-authorization may be approved when ALL are met for steroid agents continued on therapy:

Agamree continuation: Agamree (vamorolone): re-authorization up to 12 months when documentation shows continued clinical response; quantity limit remains 9 grams (225 ml) per 30 dayscontinued clinical response; 9 grams/30 days

Initial authorization updated to 12 months in 2026

Emflaza/Jaythari/Pyquvi continuation: Emflaza (deflazacort), Jaythari (deflazacort), Pyquvi (deflazacort): re-authorization up to 12 months when documentation shows continued clinical response (improvement or stable muscle strength vs baseline and/or diminished loss of muscle strength)continued clinical response

Trial of generic deflazacort required prior to branded products

Generic deflazacort continuation: Generic deflazacort: re-authorization per standard pharmacy benefit when continued clinical response documentedcontinued clinical response

Adequate trial defined as 3 continuous months

Genetic testing confirming DMD and specific mutation amenable to the indicated exon skipping therapy (document exon number).
Baseline 6-minute walk test (6MWT) distance for ambulatory individuals (record required for initial review).
Time-to-stand (when required by specific product criteria) and other functional measures as applicable.
Medication history including corticosteroid regimen and duration (stable dose length), prior trials of prednisone and generic deflazacort where applicable.

Documentation Required

Ambulatory Documentation Required for Re-authorization

Ambulatory status documentation is required for re-authorization. Continued coverage for exon-skipping IV therapies is contingent on preserved ambulation per product-specific criteria.

Re-authorization must include current documentation that the individual can ambulate (with or without permitted assistance) and meets the product-specific ambulatory thresholds (e.g., ability to complete 6MWT ≥250 meters or time-to-stand thresholds where specified).
If continued ambulation is a criterion for the specific drug, documentation must show ambulation without the use of prohibited assistive devices (wheelchair use not acceptable when noted).

Billing Rule

Benefit Assignment

Clarification of benefit assignment and billing rules for DMD therapies.

Drugs administered via IV infusion (e.g., casimersen, eteplirsen, golodirsen, vitolarsen) are managed under the medical benefit and are subject to site-of-service medical necessity review.
Self-administered or outpatient pharmacy-dispensed oral agents (e.g., deflazacort products, prednisone, vamorolone when applicable) are managed under the pharmacy benefit.
Refer to plan-specific benefit design for any variations or exceptions (e.g., Alaska fully-insured members).

Prior Authorization

Authorization Duration Limits

Initial authorizations are generally approved for up to 12 months; Elevidys (one-time gene therapy) has a different authorization expectation. Re-authorization requires documentation of clinical benefit and ongoing eligibility per product criteria.

Initial authorization duration: up to 12 months for most DMD pharmacologic therapies.
Elevidys (gene therapy) is a one-time administration — authorization considerations differ from chronic IV exon-skipping therapies.
Re-authorization requires submission of updated functional measures, ambulatory status, and confirmation of continued benefit per the specific drug's continuation criteria.

Documentation Required

Corticosteroid Trial Requirement for Deflazacort Products

For branded deflazacort products, an adequate trial of generic deflazacort is required prior to coverage of the branded product per policy update effective 2024-05-01. Documentation of trial and reason for failure or intolerance must be provided.

Documented adequate trial of generic deflazacort with dates and outcomes.
If generic deflazacort was not tolerated or failed, include clinical rationale and supporting documentation to justify branded deflazacort coverage.

Billing Rule

Site-of-Service Medical Necessity Criteria

Site-of-service medical necessity criteria are applied to medical benefit reviews; Alaska fully-insured members are excluded from these SOS criteria per Alaska HB 226. Clinical exceptions such as cytokine release syndrome (CRS) and CRS-related admission criteria are specified in the SOS rules.

Alaska fully-insured member exception: SOS Medical Necessity criteria do NOT apply — refer to infusion and injection drug Medical Necessity criteria only.
CRS exception: hospital-based outpatient or inpatient setting may be medically necessary when CRS is grade 3–4 with specified vital sign and respiratory support criteria and inpatient admission is planned.
Other SOS exceptions include unstable cardiac/pulmonary conditions, unstable renal function, difficult vascular access, acute cognitive impairment, or history of severe anaphylaxis.

Prior Authorization

Non-formulary Exception Authorizations Length

Non-formulary exception authorizations may be approved for up to 12 months per plan policy. Providers should submit supporting clinical documentation for non-formulary requests.

Non-formulary exception approvals may be issued for up to 12 months.
Provide clinical rationale and supporting records to expedite review.

Documentation Required

Steroid Stabilization Requirement and Elevidys Peri-infusional Regimen

Corticosteroid stabilization is required prior to initiation of certain IV exon-skipping therapies and specific peri-infusional steroid management is required for Elevidys. Stabilization durations and peri-infusion dosing adjustments must be documented.

Steroid stabilization: many IV DMD therapies require the individual to be on a stable corticosteroid dose for at least 3 months prior to initiation; document dosing and dates.
Elevidys peri-infusional corticosteroid regimen: initiate corticosteroid at 1 mg/kg/day starting 1 day prior to infusion and continue the increased dose for 60 days post-infusion (document start date, dose, and taper plan).
Include documentation of prior steroid trials (prednisone, generic deflazacort) and any intolerance or failures as applicable.

Re-authorization coverage criteria updated to require documentation that the individual is ambulatory without needing an assistive device for Amondys 45, Duvyzat, Exondys 51, Viltepso, and Vyondys 53 (added 12/01/25; effective March 4, 2026 following 90-day provider notification).

2025-12-01added

Added coverage criteria for Pyquvi (deflazacort) and Jaythari (deflazacort), and an appendix with examples of DMD gene mutations amenable to exon 45, 51, and 53 skipping (interim review approved Nov 11, 2025; effective March 4, 2026 for some changes).

2025-07-28safety_update

FDA removed voluntary hold for Elevidys for ambulatory patients after investigation of deaths related to acute liver failure; safety communications and related actions occurred in June–July 2025 (context for gene therapy safety considerations).

2025-02-01clarified

Clarified that Site of Service Medical Necessity criteria does not apply to Alaska fully-insured members pursuant to Alaska HB 226 and that medications listed are subject to FDA dosing/administration prescribing information.

2024-11-01revised

Elevidys age requirement updated from '4 through 5 years' to '4 years or older' and clarified that use in non-ambulatory individuals is investigational (history entry reflected in 2024 updates).

2020-04-01historical_revision

Steroid stabilization duration previously reduced from 6 months to 3 months (2020 P&T Committee change); later drug-specific durations adjusted in Part 1 criteria.

2017-01-01policy_start

Policy originated (new policy approved December 13, 2016) and subsequently updated over multiple years to add drugs, site-of-service criteria, and coding changes.