Modifiedsierra health and lifePolicy 2026D0095J

Viltolarsen (Viltepso) for Duchenne muscular dystrophy

Policy governing medical benefit coverage criteria for viltolarsen (Viltepso) to treat Duchenne muscular dystrophy (DMD) in patients with DMD gene mutations amenable to exon 53 skipping; applies to the payer's commercial and individual exchange plans.

Policy Summary

Payersierra health and life

PolicyViltolarsen (Viltepso) for Duchenne muscular dystrophy

Policy CodePolicy 2026D0095J

Change TypeRevised coverage criteria — prohibited concurrent use with givinostat

Effective DateMay 1, 2026

Next Review Date

Key ActionObtain prior authorization with medical records confirming a DMD mutation amenable to exon 53 skipping and required baseline functional assessments.

SourceLink

POLICY UPDATE CHANGES

Revised coverage criteria; added criterion requiring Viltepso will not be used concomitantly with Duvyzat (givinostat).

Transferred content to shared policy template that applies to both UnitedHealthcare Commercial and Individual Exchange benefit plans.

Added Application section to indicate policy applies to UnitedHealthcare Commercial benefit plans and Individual Exchange benefit plans.

8-10%Proportion amenable to exon 53

≤12 moInitial auth duration

≤12 moReauth duration

J1427HCPCS code

Trek Health ingests and normalizes Transparency in Coverage data and payer policy updates to give provider organizations a clear view of how commercial reimbursement behaves across markets, payers, and services. Our platform transforms raw payer disclosures into structured intelligence that supports contract evaluation, payer negotiations, and service line strategy. By combining market benchmarks with ongoing policy visibility, Trek helps teams identify variability, risk, and opportunity in commercial reimbursement. The result is faster insight, stronger negotiating positions, and more informed financial decisions.

Coverage Criteria for Viltolarsen (Viltepso)

Initial Therapy Criteria

Covered when ALL of the following are met (Initial therapy):

Initial Therapy Core Requirements

Baseline ambulatory function (one of): Either 6-Minute Walk Test (6MWT) ≥ 300 meters while walking independently prior to beginning therapy; OR patient is ambulatory without an assistive device AND one of: NSAA > 17 OR time to rise (TTR) < 7 seconds.6MWT >= 300 m OR NSAA > 17 OR TTR < 7 s
6MWT must be performed without side-by-side assist or assistive devices.
Gene therapy history: Either patient has not previously received gene therapy for DMD (e.g., Elevidys) OR patient has previously received gene therapy AND there is submission of medical records documenting a clinically meaningful functional decline (e.g., loss of a motor milestone) since gene replacement therapy.
If prior gene therapy, objective documentation of decline is required.

Continuation Therapy Criteria

Covered when ALL of the following are met (Continuation therapy):

Continuation Therapy Core Requirements: Submission of medical records confirming the patient remains ambulatory without needing an assistive device; Viltepso will not be used concomitantly with Duvyzat (givinostat) or other exon skipping therapies; prescribed by, or in consultation with, a neurologist with expertise in the treatment of DMD; dosing in accordance with the U.S. FDA–approved labeling; reauthorization for no more than 12 months.

Continuation requires ongoing ambulatory status without assistive devices.

Initial Coverage Criteria

Covered when ALL of the following are met (informed by clinical evidence and FDA indication):

FDA indication: Patient has genetically confirmed DMD with a mutation amenable to exon 53 skipping (Viltepso FDA indication; accelerated approval based on increased dystrophin).

See FDA accelerated approval information and indication.

Clinical evidence of potential benefit: Clinical trials demonstrated increased dystrophin and functional improvements versus historical comparator for timed tests (TTSTAND, TTRW) with significant differences beginning as early as week 37 and persisting through week 205; RACER53 is an ongoing randomized phase 3 trial in ambulatory boys age 4–8 years.

Efficacy and trial design details described in clinical evidence.

No concomitant givinostat: Viltepso will not be used concomitantly with Duvyzat (givinostat).

Operational revision added prohibition of concurrent use with givinostat.

Unproven Indications

Unproven/Not Medically Necessary: Use of viltolarsen (Viltepso) for forms of muscular dystrophy other than DMD amenable to exon 53 skipping (e.g., Becker muscular dystrophy) is considered unproven and not medically necessary.

Viltepso is indicated only for DMD patients with mutations amenable to exon 53 skipping.

Concomitant use is prohibited: Viltepso will not be used concomitantly with Duvyzat (givinostat) or other exon skipping therapies for DMD (for example, Amondys/casimersen, Exondys 51/eteplirsen, Vyondys 53/golodirsen). This prohibition is an explicit coverage requirement and may result in denial of coverage if documentation shows concurrent use.

Viltolarsen has not been studied in DMD that is not amenable to exon 53 skipping and has not been studied in other forms of muscular dystrophy (e.g., Becker muscular dystrophy). The FDA indication for Viltepso is limited to patients with a confirmed DMD gene mutation amenable to exon 53 skipping, and clinical trial enrollment and ongoing studies focus specifically on this population.

Use of viltolarsen for muscular dystrophies that are not amenable to exon 53 skipping (for example, Becker muscular dystrophy) is considered unproven and not medically necessary and therefore is excluded from coverage.

Requests for Viltepso in patients without documentation of a confirmed exon 53–amenable DMD mutation lack supporting clinical evidence and fall outside the FDA indication; such use is not supported by the available studies and may be denied.

Applicable Billing and Diagnosis Codes

Applicable HCPCS CodesHCPCS

J1427

Injection, viltolarsen, 10 mg.

Applicable Diagnosis CodesICD-10

G71.01

Duchenne or Becker muscular dystrophy

Provider Requirements, Prior Authorization, and Denial Conditions

Prior Authorization

Prior Authorization Required

Prior authorization required — submission of medical records confirming diagnosis, mutation amenable to exon 53 skipping, baseline assessments.

Required documentation: chart notes, laboratory values, genetic test results confirming DMD mutation amenable to exon 53 skipping
Baseline functional assessments: 6-Minute Walk Test (6MWT), North Star Ambulatory Assessment (NSAA), time to rise (TTR) as applicable
Authorization duration: initial authorization up to 12 months

Note

Policy Application

Prior authorization expected consistent with UnitedHealthcare Medical Benefit Drug Policy application to Commercial and Individual Exchange benefit plans.

Application: UnitedHealthcare Commercial and Individual Exchange benefit plans

Background on Duchenne Muscular Dystrophy and Exon Skipping

Duchenne muscular dystrophy (DMD) is an X-linked progressive muscle-wasting disorder caused by loss-of-function variants in the DMD gene that result in markedly reduced dystrophin levels. DMD leads to early progressive weakness with respiratory and cardiac complications; viltolarsen is an antisense oligonucleotide designed to skip exon 53 of dystrophin pre-mRNA to produce an internally truncated but functional dystrophin protein and is indicated for patients with mutations amenable to exon 53 skipping.

Key Definitions

inv-21: Exon 53 skipping — definition of the molecular approach using antisense oligonucleotides

DefinitionA molecular approach using antisense oligonucleotides to bind to exon 53 of dystrophin pre-mRNA, causing exclusion of exon 53 during mRNA processing to allow production of an internally truncated but functional dystrophin protein.

Therapeutic modalityAntisense oligonucleotide (Viltepso/viltolarsen) designed to target exon 53 of dystrophin pre-mRNA.

Intended effectRestore reading frame in selected DMD mutations to increase dystrophin expression in skeletal muscle.

Population proportionApproximately 8%–10% of DMD patients have out-of-frame deletion mutations amenable to exon 53 skipping.

inv-22: Amenable to exon 53 skipping — definition regarding confirmed DMD gene mutation targetable by exon 53 skipping