ModifiedUnitedHealthcarePolicy CSPA2026D0095I

Viltepso Pa Cs

State-specific (Pennsylvania) UnitedHealthcare medical benefit drug policy governing coverage criteria, initial and continuation authorization, and applicable coding for Viltepso (viltolarsen) for treatment of Duchenne muscular dystrophy amenable to exon 53 skipping.

Policy Summary

PayerUnitedHealthcare

PolicyViltepso Pa Cs

Policy CodePolicy CSPA2026D0095I

Change TypeRevised coverage criteria — added concomitant use exclusion with Duvyzat (givinostat)

Effective DateJun 1, 2026

Next Review Date

Key ActionSubmit medical records confirming the DMD gene mutation is amenable to exon 53 skipping and document baseline and ongoing standardized motor function assessments by a neurologist; avoid concomitant use with Duvyzat or other exon skipping therapies.

SourceLink

POLICY UPDATE CHANGES

Revised coverage criteria to add a requirement that Viltepso will not be used concomitantly with Duvyzat (givinostat).

1Covered Indication (DMD amenable to exon 53 skipping)

12 monthsInitial Authorization Duration

12 monthsReauthorization Duration

J1427Primary HCPCS Code

Coverage Summary

Coverage stance: covered_with_criteria for Viltepso (viltolarsen) when used to treat Duchenne muscular dystrophy (DMD) with a DMD gene mutation amenable to exon 53 skipping. Scope: State-specific (Pennsylvania) UnitedHealthcare medical benefit drug policy governing coverage criteria, initial and continuation authorization, and applicable coding for Viltepso. Subject: Viltepso (viltolarsen) for Duchenne muscular dystrophy (PA only). Effective/last review date: Effective: 2026-06-01; Last review: 2026-06-01. Approval context: indication granted under accelerated approval based on increased dystrophin expression. Approximately 8%–10% of DMD patients have mutations amenable to exon 53 skipping.

Key Coverage Stats

Primary HCPCS CodeJ1427

Covered Indication (count)1

Initial Authorization Duration12 months

Reauthorization Duration12 months

Initial Therapy

Covered when ALL of the following are met:

ALL of the following

Diagnosis of Duchenne muscular dystrophy by, or in consultation with, a neurologist with expertise in the diagnosis of DMD
Submission of medical records (e.g., chart notes, laboratory values) confirming the mutation of the DMD gene is amenable to exon 53 skipping
Submission of medical records documenting a baseline evaluation, including a standardized assessment of motor function by a neurologist with experience in treating Duchenne muscular dystrophy, prior to beginning Viltepso therapy
ONE of the following
- Patient has not previously received gene therapy (e.g., Elevidys (delandistrogene moxparvovec-rokl)) for the treatment of DMD

Continuation Therapy (Reauthorization)

Unproven / Not Medically Necessary

Applicable Codes

Covered HCPCS (drug administration/product)HCPCSCovered

J1427

Injection, viltolarsen, 10 mg

Relevant Diagnosis Codes (indication reference)ICD-10

G71.01

Duchenne or Becker muscular dystrophy

Provider Actions & Prior Authorization

Documentation Required

Genotype confirmation required

Submit medical records (e.g., chart notes, laboratory values) confirming the DMD gene mutation is amenable to exon 53 skipping with the initial request. Documentation of genotype confirmation is required for coverage determination.

Documentation Required

Baseline and ongoing motor function assessments

Provide medical records documenting a baseline standardized motor function assessment performed by a neurologist experienced in treating Duchenne muscular dystrophy prior to starting Viltepso. For reauthorization, submit standardized assessments documenting continued clinical benefit, also performed by a neurologist experienced in DMD.

Clinical Evidence

up to 192 wksLong-term extension follow-up (participants enrolled from Phase 2 into LTE up to 192 weeks; analyses reported through ~205 weeks)

TTSTAND p<0.05TTSTAND improvement significant from week 73 through week 205 vs CINRG DNHS

TTRW significantTTRW stabilization then significant slowing of decline vs CINRG DNHS beginning week 73 and maintained to week 205

Evidence summary: Viltepso received accelerated approval based on increased dystrophin in skeletal muscle. In the Phase 2 study, high-dose viltolarsen (80 mg/kg) produced a mean increase in dystrophin by Western blot from 0.6% to ~5.9% of normal at Week 25 (mean change ~5.3%, p=0.01). Functional comparisons versus a matched natural history cohort over 25 weeks showed improvement or stabilization in timed function tests, notably a +28.9 m change in the 6-minute walk test versus −65.3 m in controls. Long-term extension data (follow-up out to ~192–205 weeks) demonstrated significant improvements or stabilization on TTSTAND and TTRW versus the CINRG DNHS comparator and reported that viltolarsen was well tolerated.

Background & Definitions

Background: Viltolarsen is an antisense oligonucleotide designed to bind to exon 53 of dystrophin pre-mRNA, resulting in exclusion of this exon during mRNA processing to enable production of an internally truncated dystrophin protein in patients with mutations amenable to exon 53 skipping. Approximately 8% to 10% of DMD patients have out-of-frame deletion mutations amenable to exon 53 skipping. The FDA indication was granted under accelerated approval based on an increase in dystrophin observed in some patients treated with Viltepso.

Definitions: Amenable to exon 53 skipping: A confirmed mutation of the DMD gene (out-of-frame deletion) for which viltolarsen exon 53 skipping can restore the reading frame to produce truncated dystrophin.

Revision History

2026-06-01policy_modifiedLatest

Revised coverage criteria to add a requirement that Viltepso will not be used concomitantly with Duvyzat (givinostat).

Archived: CSPA2025D0095Hpolicy_archived

Archived previous policy version CSPA2025D0095H.

Policy Summary

PayerUnitedHealthcare

PolicyViltepso Pa Cs

Policy CodePolicy CSPA2026D0095I

Change TypeRevised coverage criteria — added concomitant use exclusion with Duvyzat (givinostat)

Effective DateJun 1, 2026

Next Review Date

SourceLink

On This Page

If patient previously received gene therapy

Patient has previously received gene therapy (e.g., Elevidys (delandistrogene moxparvovec-rokl)) for the treatment of DMD
Submission of medical records (e.g., chart notes, laboratory values) documenting a clinically meaningful functional decline resulting from loss of muscle strength/motor ability (e.g., loss of a motor milestone) since receiving gene replacement therapy

Viltepso will not be used concomitantly with Duvyzat (givinostat)

Viltepso will not be used concomitantly with other exon skipping therapies for DMD (e.g., Amondys (casimersen), Exondys 51 (eteplirsen), Vyondys 53 (golodirsen))

Prescribed by, or in consultation with, a neurologist with expertise in the treatment of DMD

Dosing is in accordance with the United States Food and Drug Administration approved labeling

Prior Authorization

Prescriber specialty and consultation requirement

Prescriptions must be by, or in consultation with, a neurologist with expertise in diagnosing and treating DMD. Initial authorization and reauthorization periods are limited to no more than 12 months.

Denial Risk

Concomitant therapy exclusion

Claims may be denied if Viltepso is used concomitantly with Duvyzat (givinostat) or with other exon skipping therapies for DMD (e.g., casimersen, eteplirsen, golodirsen). Do not combine therapies; coverage requires Viltepso not be used concomitantly with these agents.

Billing Rule

Dosing per FDA label

Dosing must follow the United States Food and Drug Administration approved labeling to qualify for coverage. Use HCPCS code J1427 (Injection, viltolarsen, 10 mg) for billing.