Duchenne Muscular Dystrophy (DMD) drug therapy coverage
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Pharmacy management drug policy governing coverage and prior authorization criteria for multiple DMD therapies (including Agamree, Amondys 45, Duvyzat, Exondys 51, Viltepso, Vyondys 53) for Univera Healthcare lines of business.
Multiple revisions were made with update entries spanning 2024–2026, including a revision on 06/17/2026.
Drug-Specific Coverage Criteria
Agamree
Covered when ALL of the following are met
See chunks 15-16 for details
Amondys 45
Covered when ALL of the following are met
Continued approval requirements specified in continuation criteria (see continuation block)
Duvyzat
Covered when ALL of the following are met
See chunk 19 for laboratory and exclusion criteria
Amondys 45 - Initial and Continuation Criteria
Covered when ALL of the following are met
Continued approval for non-new starts requires documentation every 6 months that patient remains ambulatory with unassisted 6MWT >=100 m, LVEF >40%, FVC >=50% predicted, and <5% annual decline in FVC% (progress notes/test results must be dated after most recent drug approval and within 3 months).
Duvyzat - Initial and Continuation Criteria
Covered when ALL of the following are met
See chunk 19
Exondys 51 - Initial and Continuation Criteria
Covered when ALL of the following are met
Continued approval for non-new starts requires documentation every 6 months that patient remains ambulatory with unassisted 6MWT >=100 m, LVEF >40%, FVC >=50% predicted, and <5% annual decline in FVC%.
Viltepso - Initial and Continuation Criteria
Covered when ALL of the following are met
Continued approval for non-new starts requires documentation every 6 months that patient remains ambulatory with unassisted 6MWT >=100 m, LVEF >40%, FVC >=50% predicted, and <5% annual decline in FVC%.
Vyondys 53 - Initial and Continuation Criteria
Covered when ALL of the following are met
Continued approval for non-new starts requires documentation every 6 months that patient remains ambulatory with unassisted 6MWT >=100 m, LVEF >50%, FVC >=50% predicted, and <5% annual decline in FVC%.
Emflaza / generic deflazacort / Jaythari / Kymbee / Pyquvi - Initial and Continuation Criteria
Covered when ALL of the following are met
See chunks 21-23 for dosing/quantity rounding and pediatric exceptions
Concurrent use of exon-skipping therapies is not allowed: patients must not be treated with more than one exon-skipping IV product at the same time. In addition, certain exon‑skipping products will be denied if the patient has previously received Elevidys; specifically, Amondys 45, Exondys 51, Viltepso, and Vyondys 53 are not approved for patients previously treated with Elevidys. These concurrent‑use and prior‑treatment exclusions are explicit criteria for medical‑benefit authorization and must be documented in the medical record or prior‑auth submission.
Duvyzat is explicitly excluded from use in combination with exon‑skipping therapies; requests for Duvyzat must demonstrate it is not being given concurrently with any exon‑skipping agent and that there is no prior Elevidys exposure.
Providers should ensure documentation of prior therapies and current concurrent medications is included in each authorization request to allow accurate application of these exclusions.
Patients with prior treatment with Elevidys are ineligible for approval of multiple exon‑skipping IV products. The policy specifies that Amondys 45, Exondys 51, Viltepso, and Vyondys 53 will not be approved for use in patients who have previously received Elevidys. This prior‑treatment exclusion must be confirmed in the clinical history submitted with the prior authorization request.
Duvyzat will not be covered if administered in combination with exon‑skipping therapies or when there is prior use of Elevidys. The policy states: “Duvyzat will not be covered in combination with exon‑skipping therapies or prior use of Elevidys.” Ensure lab and medication history submitted with the request document absence of concurrent exon‑skipping therapy and no prior Elevidys exposure.
Oral deflazacort products and alternatives — Jaythari, Kymbee, and Pyquvi — are only considered when there is a documented, clinically significant hypersensitivity or serious adverse reaction attributed to an inactive ingredient in Emflaza or generic deflazacort that is not present in the requested product. For prior authorization, include documentation of the hypersensitivity or adverse reaction and evidence that the offending inactive ingredient is absent from the alternative product.
Coverage of listed HCPCS and other codes is contract dependent. The policy notes that eligibility for reimbursement is based upon the member's subscriber contract and that codes may not be covered under all circumstances. Providers should verify benefit/contract rules prior to submitting claims and include any contract‑required documentation with prior authorization requests.
Also note that some member contracts may exclude medical‑infused drugs from pharmacy benefits or otherwise limit coverage for infusion services; refer to the member’s specific contract language when determining eligibility.
IV exon‑skipping therapies (Amondys 45, Exondys 51, Vyondys 53, Viltepso) are intended for ambulatory patients only and will not be approved for those who have lost ambulation or whose cardiac or pulmonary testing indicates severe decompensation. Specifically, these agents will not be approved for patients who have lost ambulation or who have left ventricular ejection fraction (LVEF) or forced vital capacity (FVC% predicted) results that indicate severe cardiac or respiratory compromise, because pivotal trials evaluated these drugs in ambulatory populations. Verify baseline ambulatory status and recent LVEF and FVC% results when submitting authorization requests.
Initial Authorization Requirements
Initial therapy — drug-specific initial authorization criteria
Initial therapy authorization criteria (drug-specific)
See product-specific criteria blocks for exact thresholds and exclusions
Initial therapy requirements — product-specific initiation rules and limits
Product-specific initiation rules and limits
See chunks 18,24,25,27,29,19,22 for per-product thresholds and limits
Recertification and Continued Approval
Amondys 45 continuation
Amondys 45 continuation requirements
Progress notes/test results must be dated after most recent drug approval and within 3 months
Agamree and Duvyzat continuation
Agamree and Duvyzat continuation requirements
See chunk 15
See chunk 19
Continuation therapy requirements — criteria for continued approval after initial period
Criteria for continued approval after initial period, per product
Progress notes/test results must be dated after the most recent drug approval and within 3 months of the continuation period request.
See chunks 19 and 21-23
HCPCS / Clinical Test Requirements
Prior Authorization, Documentation, and Denial Triggers
Agamree prior authorization
Agamree prior authorization: prior authorization required. Provider must document a trial of prednisone or deflazacort for ≥6 months and record at least one specified intolerable adverse effect (e.g., crossing major height-percentile curves, vertebral fractures, reductions in bone-formation/turnover biomarkers, unmanageable diabetes/hypertension, or side effects requiring hospitalization). Initial approval is 12 months; dose and quantity limits apply.
- Trial of prednisone or deflazacort ≥6 months (documentation required)
- Document one intolerable adverse effect as listed (height-percentile decline, vertebral fracture, bone biomarker changes, uncontrolled diabetes/hypertension, or hospitalization for side effects)
- Initial approval: 12 months; dose ≤6 mg/kg/day and ≤300 mg/day for patients >50 kg; quantity limit 100 mL/30 days
Amondys 45 prior authorization
Amondys 45 prior authorization: provider must document a confirmed DMD mutation amenable to exon‑45 skipping, stable corticosteroid use for ≥6 months (or documented reason not to use), kidney function testing prior to initiation, and that the patient is not concurrently on another exon‑skipping therapy or previously treated with Elevidys. Initial approval for new starts is 3 months.
- Confirmed exon 45–amenable DMD mutation
- Stable corticosteroids ≥6 months prior or documented reason not to be on corticosteroids
- Kidney function testing prior to starting therapy
- Not concurrently on another exon‑skipping therapy; no prior Elevidys treatment
- Initial approval: 3 months; dose ≤30 mg/kg once weekly
Duvyzat prior authorization
Duvyzat prior authorization: provider must confirm age ≥6, a DMD diagnosis with confirmed mutation, that the patient is ambulatory at initiation, and documentation of baseline corticosteroid use for 6 months with intention for ongoing concomitant use. Baseline laboratory results within the past 3 months are required to confirm eligibility.
- Age ≥6 years and specialist prescriber
- Ambulatory at initiation
- Baseline corticosteroids for 6 months with intent to continue
- Laboratory results within past 3 months to document platelet/WBC/hemoglobin, fasting triglycerides, and baseline QTcF
- Initial approval: 12 months; dose/quantity limits apply
PA required for IV exon-skipping therapies
Prior authorization required for all IV exon‑skipping therapies administered under the medical benefit. Providers must submit PA requests for the HCPCS codes listed in the policy and meet drug‑specific initiation criteria (e.g., ambulatory status, 6MWT thresholds, cardiac/pulmonary stability).
- IV exon‑skipping therapies are medical‑benefit infusions (Amondys 45, Exondys 51, Viltepso, Vyondys 53)
- Submit PA using the HCPCS codes listed in policy and provide required clinical documentation per product
Prior authorization for DMD HCPCS codes
Prior authorization applies to the specified HCPCS drug codes for DMD exon‑skipping infusions; coverage is contract dependent so providers should verify member benefits before billing. The listed HCPCS codes correspond to the products noted in the policy.
Agamree and Duvyzat corticosteroid prior/concurrent use requirements
Agamree and Duvyzat require prior or ongoing corticosteroid therapy documentation: Agamree requires a prior prednisone or deflazacort trial of ≥6 months (with intolerance criteria documented if applicable); Duvyzat requires baseline corticosteroids for 6 months with intent for ongoing concomitant use.
- Agamree: documentation of prednisone/deflazacort trial ≥6 months and listed intolerable adverse effects
- Duvyzat: baseline corticosteroid use for 6 months and plan to continue concomitantly
Utilization management and step therapy
Utilization management and step‑therapy may be applied at recertification: providers should expect that recertification reviews can require trials of more cost‑effective alternatives (e.g., generics, biosimilars) as they become available and that step therapy may be imposed in addition to LCD/NCD requirements.
- Recertification may require trial of cost‑effective alternatives
- Step therapy may be imposed in addition to NCD/LCD requirements
Documentation required for prior prednisone/deflazacort trial and listed adverse effects
Documentation required for prior prednisone/deflazacort trial: providers must submit documentation of the ≥6‑month steroid trial and evidence of the listed intolerable adverse effects (e.g., decline in height percentile crossing major curves, vertebral fractures, reductions in bone‑formation/turnover biomarkers, unmanageable diabetes/hypertension, or side effects requiring hospitalization).
- Document steroid trial duration (≥6 months) and specific adverse‑effect evidence per policy (height‑percentile crossing, vertebral fractures, osteocalcin/PINP/CTX1 changes, uncontrolled diabetes/hypertension, hospitalization for side effects)
Documentation must indicate kidney function testing prior to starting Amondys 45
For Amondys 45 initiation and continuation requests, documentation must indicate kidney function testing prior to starting therapy and progress notes/test results dated after the most recent drug approval and within 3 months for continuation requests.
- Kidney function testing results prior to initiation
- Progress notes/test results must be dated after most recent drug approval and within 3 months for continuation requests
Duvyzat laboratory requirements — labs within past 3 months
Duvyzat laboratory requirements: providers must submit laboratory results taken within the past 3 months to document platelet, white blood cell, and hemoglobin counts; fasting triglycerides; and baseline QTcF prior to approval.
- Platelet, WBC, and hemoglobin counts within normal limits (labs within past 3 months)
- Fasting triglycerides ≤300 mg/dL (fasting measurement)
- Baseline QTcF ≤450 msec (mean of 3 consecutive readings)
Required supporting documentation — progress notes, diagnostics, genetic testing, objective measures
Required supporting documentation for any PA request includes clinical progress notes, diagnostic testing, laboratory test results, genetic testing, and objective measures of function; recertification must demonstrate ongoing benefit or stabilization.
- Progress notes documenting previous treatments and treatment history
- Diagnostic testing and laboratory results
- Genetic testing or biomarker results and other objective/subjective measures of clinical benefit
- Recertification requires evidence of improvement or stability and that continued use remains medically necessary
Baseline and follow-up testing — cardiac, pulmonary, 6MWT within 3 months
Baseline and follow‑up testing must be submitted for IV exon‑skipping therapies: provide recent (within 3 months) cardiac (LVEF), pulmonary (FVC% predicted), and unassisted 6‑minute walk test (6MWT) results for initiation and continuation as specified by product criteria.
- Baseline unassisted 6MWT thresholds by product and recent 6MWT within 3 months (e.g., Amondys 45 baseline ≥300 m; continuation ≥100 m)
- LVEF per product requirement (e.g., >40% for many products; Vyondys 53 >50%)
- FVC% predicted ≥50% and not on supplemental oxygen; <5% annual decline required for continuation
Documentation and coding currency — providers should monitor coding updates
Providers should monitor coding updates and read the policy carefully: eligibility for reimbursement and the applicability of listed codes may change as AMA/CMS codes are updated more frequently than policy revisions.
- Codes may not be covered under all circumstances; verify coverage per member subscriber contract
- Monitor AMA and CMS coding updates that may post more frequently than policy updates
Duvyzat will not be covered in combination with exon-skipping therapies or prior Elevidys
Duvyzat will not be covered in combination with exon‑skipping therapies or in patients with prior Elevidys treatment; do not submit PA requests for concurrent exon‑skipping use or for patients previously treated with Elevidys.
- Duvyzat is excluded when used in combination with exon‑skipping therapies
- Prior Elevidys treatment excludes Duvyzat coverage
Amondys 45 will not be approved for patients previously treated with Elevidys
Amondys 45 will not be approved for patients previously treated with Elevidys; providers must document no prior Elevidys treatment when requesting PA for Amondys 45.
- Verify and document no prior Elevidys treatment before submitting PA for Amondys 45
Laboratory and ECG exclusion triggers for Duvyzat (low CBC, triglycerides >300, QTcF >450 msec)
Laboratory and ECG exclusion triggers for Duvyzat: patients with platelet/WBC/hemoglobin counts below the lower limit of normal, fasting triglycerides >300 mg/dL, or baseline QTcF >450 msec (or additional torsades risk factors) are ineligible for coverage.
- Platelet, WBC, or hemoglobin counts less than lower limit of normal — documentation required
- Fasting triglycerides >300 mg/dL exclude coverage
- Baseline QTcF >450 msec (mean of 3 readings) or history of torsades risk factors excludes coverage
Missing documentation may result in denial
Missing clinical documentation may result in denial: PA requests (initial and recertification) require submission of supporting clinical documentation unless otherwise specified; lack of documentation may lead to denial.
- Clinical documentation is required for each request (progress notes, labs, diagnostics, genetic testing, objective measures)
- Recertification must show ongoing benefit/stability
Reimbursement eligibility and code coverage dependent on member contract
Reimbursement eligibility and coverage of codes depend on the member's subscriber contract; providers must verify benefits before billing or assuming coverage.
- Eligibility for reimbursement is based on the member's subscriber contract
- Codes listed may not be covered under all circumstances
Step Therapy and Prior Treatment Requirements
| Requirement | Details |
|---|---|
| Documentation of prior corticosteroid therapy | |
| Agamree: trial of prednisone or deflazacort for ≥6 months (documentation required) or evidence of intolerable adverse effects as specified (e.g., crossing height percentiles, vertebral fractures, reduced bone biomarkers, unmanageable diabetes/hypertension, or hospitalization for steroid-related adverse events). Duvyzat: must be on baseline corticosteroids for 6 months with intention for ongoing concomitant use. |
| Recertification condition | Policy implication |
|---|---|
| Requirement to consider cost-effective alternatives at recertification | |
| Recertification reviews may require trial of more cost-effective alternatives (e.g., generics, biosimilars, or other guideline-supported options) as they become available; continued approval requires documentation of ongoing benefit or stability. |
Quantity Limits by Product
Administration Setting and Benefit Determination
infusion center | hospital outpatient | office — IV exon-skipping therapies are medical benefit infusions
IV exon‑skipping therapies (Amondys 45, Exondys 51, Vyondys 53, Viltepso) are administered as IV infusions and are covered under the medical benefit; site of care (infusion center, hospital outpatient, office) may impact approval timeframe and is subject to review.
- IV exon‑skipping therapies are medical‑benefit infusions
- Site of care options: infusion center, hospital outpatient, or office; site may affect approval timeframe
home — oral agents are pharmacy benefit, self-administered
Oral agents (Duvyzat, deflazacort products, Agamree) are self‑administered at home and are covered under the pharmacy benefit; providers should submit pharmacy benefit prior authorization requests as applicable.
- Oral agents are pharmacy‑benefit, self‑administered at home
- Submit pharmacy PA requests per formulary/benefit rules
Clinical Background
Duchenne muscular dystrophy (DMD) is a progressive, X‑linked genetic disorder caused by mutations in the dystrophin gene leading to muscle degeneration and loss of function. Management includes corticosteroids and multidisciplinary supportive care; several therapies for DMD use antisense oligonucleotide exon‑skipping to restore a truncated dystrophin protein for patients with specific exon‑amenable mutations. Many exon‑skipping approvals are on an FDA accelerated approval basis using dystrophin as a surrogate endpoint, and confirmatory clinical benefit for some products remains under investigation. Clinical eligibility for the therapies covered by this policy is based on product‑specific mutation amenability, ambulatory status, and cardiac/pulmonary stability; refer to individual drug criteria for exact thresholds and testing requirements.
Key Terms and Definitions
Policy Revision Timeline
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