CurrentEmblemHealthPolicy MG.MM.PH.321

Viltepso (viltolarsen) for Duchenne muscular dystrophy

Clinical coverage criteria, dosing, and authorization requirements for Viltepso (viltolarsen) for members with Duchenne muscular dystrophy (DMD) amenable to exon 53 skipping. Applies to EmblemHealth/ConnectiCare members and treating providers requesting authorization.

Policy Summary

PayerEmblemHealth

PolicyViltepso (viltolarsen) for Duchenne muscular dystrophy

Policy CodePolicy MG.MM.PH.321

Change TypeMaterial updates to dosing, renewal, monitoring, and concomitant therapy exclusions

Effective Date

Next Review DateApr 2, 2025

Key ActionObtain prior authorization and submit documentation demonstrating diagnosis, mutation amenable to exon 53 skipping, baseline objective measures, and periodic monitoring including renal markers for renewal.

SourceLink

POLICY UPDATE CHANGES

Updated dosing limits.

Added limitation excluding concomitant therapy with delandistrogene moxeparvovec-rokl.

Added requirement to measure serum cystatin C, urine dipstick, and urine protein-to-creatinine ratio prior to and periodically during treatment.

Renewal criteria changed to require response to therapy compared to pretreatment baseline in one or more objective measures (e.g., dystrophin level, timed function tests, NSAA, FVC).

6 monthsinitial/renewal authorization length

80 mg/kgmaximum dosing limit

J1427applicable HCPCS/J-code

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NDC 73292-0011-01single-dose vial

Coverage Criteria for Viltepso (viltolarsen)

Initial Approval

Covered when ALL of the following are met

Initial Approval Criteria: A. Medication is being prescribed by or in consultation with a neurologist who specializes in treatment of Duchenne muscular dystrophy (DMD); AND B. Patient has been diagnosed with Duchenne muscular dystrophy (DMD); AND C. Documentation has been provided confirming mutation of the DMD gene amenable to exon 53 skipping; AND D. Member must have been on a stable dose of corticosteroids for at least 3 months (unless contraindicated); AND E. Patient retains meaningful voluntary motor function (e.g., patient is able to speak, manipulate objects using upper extremities, ambulate, etc.); AND F. Patient is receiving physical and/or occupational therapy; AND G. Baseline documentation of ONE or more of the following: i. Dystrophin level; ii. Timed function tests (e.g., 6-minute walk test [6MWT], time to stand [TTSTAND], time to run/walk 10 meters [TTRW], time to climb 4 stairs [TTCLIMB], etc.); iii. Upper limb function (ULM) test; iv. North Star Ambulatory Assessment (NSAA) score; v. Forced Vital Capacity (FVC) percent predicted; AND H. Viltepso is not used concomitantly with other exon skipping therapies for DMD.

All subcriteria A through H must be satisfied.

Renewal

Renewal covered when ALL of the following are met

Renewal Criteria: A. Patient continues to meet INITIAL APPROVAL CRITERIA; AND B. Patient has responded to therapy compared to pretreatment baseline in one or more of the following: i. Increase in dystrophin level; ii. Improvement in quality of life; iii. Stability, improvement, or slowed rate of decline in one or more of: timed function tests (6MWT, TTSTAND, TTRW, TTCLIMB), upper limb function (ULM) test, NSAA score, FVC percent predicted; AND C. Absence of unacceptable toxicity from the drug has been documented (examples include glomerulonephritis, persistent increase in serum cystatin C, proteinuria); AND D. Viltepso is not used concomitantly with other exon skipping therapies for DMD.

Renewal interval up to 6 months.

Use of Viltepso for indications not supported by CMS-recognized compendia or acceptable peer‑reviewed literature may be deemed not approvable and therefore not reimbursable. Viltepso must not be used concomitantly with other DMD‑directed antisense oligonucleotides (for example, eteplirsen, casimersen, golodirsen) or with d elandistrogene moxeparvovec‑rokl. Renal function should be monitored during therapy; note that serum creatinine may not be a reliable measure of renal function in DMD patients. Prior to starting therapy and periodically during treatment, measure serum cystatin C, urine dipstick, and urine protein‑to‑creatinine ratio (UPCR). Patients with symptomatic cardiomyopathy are excluded from coverage for Viltepso.

There are no clinical studies demonstrating use in the geriatric population (≥65 years). Duchenne muscular dystrophy is predominantly a disease of children and young adults; therefore safety and effectiveness in older adults have not been established.

Coverage may be discontinued if the patient demonstrates disease progression while on Viltepso. Uses that are not supported by CMS‑recognized compendia or acceptable peer‑reviewed literature are considered not approvable and not reimbursable. The 4/2/2025 revision updated limitations to explicitly exclude concomitant use with d elandistrogene moxeparvovec‑rokl and added requirements for baseline and periodic measurement of serum cystatin C, urine dipstick, and UPCR during treatment.

Coding and Dosing Limits

Applicable HCPCS/J-codeHCPCSCovered

J1427

Injection, viltolarsen, 10 mg; 1 billable unit = 10 mg

Applicable NDCsNDC

73292-0011-01

Viltepso single-dose vials containing 250 mg/5 mL (50 mg/mL)

ICD-10 DiagnosesICD-10

G71.01

Duchenne or Becker muscular dystrophy

Maximum dose per week — operational dosing limit and billable units

Operational dosing limit80 mg/kg IV once weekly

Billable units per week925 billable units (9200 mg) every 7 days

Billing conversion1 billable unit = 10 mg (see HCPCS J1427)

Provider Actions and Documentation Requirements

Prior Authorization

Prior Authorization Required

Prior authorization is required for Viltepso. Initial authorization length: coverage will be provided for 6 months and may be renewed.

Prior authorization required
Initial approval length: 6 months

Denial Risk

Concomitant Therapy Prohibition

Viltepso must not be used concomitantly with other exon-skipping therapies for DMD. Additionally, concomitant use with delandistrogene moxeparvovec-rokl is explicitly excluded.

No concomitant exon-skipping therapies (e.g., eteplirsen, casimersen, golodirsen)
No concomitant delandistrogene moxeparvovec-rokl

Background

Viltepso (viltolarsen) is an antisense oligonucleotide that binds exon 53 of dystrophin pre‑mRNA and induces exon 53 skipping, enabling production of an internally truncated dystrophin protein in patients with DMD mutations amenable to exon 53 skipping. Approval was granted under an accelerated approval pathway based on increased dystrophin production; continued approval may depend on confirmatory trials. The policy revision history dated 4/2/2025 reflects updates to dosing limits and to safety monitoring and concomitant‑therapy exclusions.

Definitions

Exon 53 skipping — molecular mechanism

MechanismViltepso binds to exon 53 of dystrophin pre-mRNA causing exclusion of exon 53 during mRNA processing

ResultExon 53 skipping is intended to allow production of internally truncated dystrophin protein in patients with mutations amenable to exon 53 skipping

Regulatory noteIndication approved under accelerated approval based on increased dystrophin production; continued approval may depend on confirmatory trials

Viltepso (viltolarsen) — drug and approval status

Drug class and actionViltepso (viltolarsen) is an antisense oligonucleotide that binds exon 53 of dystrophin pre-mRNA to induce exon 53 skipping

Approval statusApproved under accelerated approval based on increased dystrophin production in skeletal muscle; continued approval may be contingent on confirmatory trials

Policy Summary

PayerEmblemHealth

PolicyViltepso (viltolarsen) for Duchenne muscular dystrophy

Policy CodePolicy MG.MM.PH.321

Change TypeMaterial updates to dosing, renewal, monitoring, and concomitant therapy exclusions

Effective Date

Next Review DateApr 2, 2025

SourceLink

On This Page

Documentation Required

Required Clinical Documentation for Authorization

Submit clinical documentation to support medical necessity. Required items include diagnosis and documentation confirming a DMD gene mutation amenable to exon 53 skipping, prescribing clinician specialty, baseline objective measures (dystrophin level, timed function tests, ULM, NSAA, FVC % predicted), evidence of stable corticosteroid use or contraindication, and monitoring labs (serum cystatin C, urine dipstick, UPCR). Failure to provide these items may prevent preauthorization or trigger post-payment review.

Diagnosis of Duchenne muscular dystrophy
Documentation confirming DMD mutation amenable to exon 53 skipping
Prescriber: neurologist specializing in DMD (or consultation)
Baseline measures: dystrophin level; timed function tests (6MWT, TTSTAND, TTRW, TTCLIMB); upper limb function (ULM); NSAA score; FVC % predicted
Documentation of stable corticosteroid dose for ≥3 months or contraindication
Monitoring labs: serum cystatin C, urine dipstick, urine protein-to-creatinine ratio (UPCR) prior to and periodically during therapy

Documentation Required

Renewal Documentation and Monitoring

For renewal, submit documentation demonstrating continued meeting of initial criteria and objective evidence of response compared to pretreatment baseline (examples: increased dystrophin level, improved quality of life, or stability/improvement/slower decline in timed function tests, ULM, NSAA, or FVC % predicted). Also document absence of unacceptable toxicity (e.g., glomerulonephritis, persistent increase in serum cystatin C, proteinuria). Renewals are granted in up to 6-month intervals.

Renewal interval: up to 6 months
Evidence of response: increase in dystrophin level; improved quality of life; stability/improvement/slowed decline in timed function tests, ULM, NSAA, or FVC % predicted
Document absence of unacceptable toxicity (e.g., glomerulonephritis, persistent rise in serum cystatin C, proteinuria)
Documentation must show continued fulfillment of initial approval criteria

Denial Risk

Documentation Submission Requirement / Unsupported Indications

Indications not supported by CMS-recognized compendia or acceptable peer-reviewed literature may be denied and are not reimbursable. Failure to submit required evidence will prevent preauthorization or may trigger post-payment review.

Unsupported indications per compendia/literature may be denied
Failure to submit required documentation prevents preauthorization or may lead to post-payment review

IndicationFor patients with genetic mutations amenable to exon 53 skipping