CurrentAnthemPolicy ING-CC-0172 - NY

Viltepso (viltolarsen) - NY Medicaid

NY Medicaid clinical coverage criteria for use of Viltepso (viltolarsen) for treatment of Duchenne muscular dystrophy with a mutation amenable to exon 53 skipping, including initial and continuation approval criteria, monitoring, quantity limits, and applicable billing codes.

Policy Summary

PayerAnthem

PolicyViltepso (viltolarsen) - NY Medicaid

Policy CodePolicy ING-CC-0172 - NY

Change TypeNew York specific policy (created)

Effective Date04/01/2022

Next Review Date

Key ActionPrior authorization requests must document confirmed DMD with an exon 53–amenable mutation, age 4–9 years, corticosteroid use, baseline kidney testing, and no concurrent exon skipping agents.

POLICY UPDATE CHANGES

New York specific policy created based on guidance released from NYSDOH.

08/20/2021 Annual Review: No changes.

08/01/2021 Administrative update to add documentation.

08/21/2020 New clinical criteria and quantity limit added for Viltepso; coding updates applied over 2020-2021.

1Covered Indication (DMD amenable to exon 53 skipping)

80 mg/kg weeklyTypical dosing

6 monthsApproval duration

Coverage Summary

NY Medicaid clinical coverage criteria for Viltepso (viltolarsen) for treatment of Duchenne muscular dystrophy with a mutation amenable to exon 53 skipping, including initial and continuation approval criteria, monitoring, quantity limits, and applicable billing codes. Policy stance: covered_with_criteria. Policy number: ING-CC-0172 - NY.

Medical Necessity Criteria

Initial therapy - Viltepso (viltolarsen)

Initial requests for Viltepso (viltolarsen) may be approved if the following criteria are met:

Initial therapy criteria

Confirmed diagnosis: Individual has a confirmed diagnosis of Duchenne muscular dystrophy (DMD)
Genetic mutation: Documentation is provided that individual has a genetic mutation that is amenable to exon 53 skipping
Age: Individual is age 4-9 years (NCT02740972) (Clemens 2020)4-9 years
Corticosteroid use: Individual is using a corticosteroid
Kidney testing: Documentation indicates kidney function testing prior to starting therapy
Recommended baseline tests: serum cystatin C, urine dipstick, urine protein-to-creatinine ratio (serum creatinine may be unreliable in DMD)
No concurrent exon skipping: Individual will not use with any other exon skipping agents for DMD (including but not limited to Vyondys 53)

Continuation therapy - Viltepso (viltolarsen)

Continuation of therapy may be approved if the following criterion is met:

Continuation criterion

Met initiation criteria: Criteria for initiation (above) were met at initiation of therapy

Exclusions / Not approved

Requests may not be approved when the criteria above are not met and for all other indications.

Exclusions

Other indications: Any indication other than DMD with a mutation amenable to exon 53 skipping
Age not eligible: Requests for individuals who do not meet the age requirement of 4-9 years4-9 years
Concurrent exon skipping: Concurrent use with other exon skipping agents for DMD

Provider Actions

Prior Authorization

Prior authorization required with clinical criteria

Initial and continuation prior authorization requests require documentation that all initial criteria are met before approval. Required documentation includes confirmation of a Duchenne muscular dystrophy (DMD) diagnosis, evidence the individual has a genetic mutation amenable to exon 53 skipping, age within the 4-9 year range, concurrent use of a corticosteroid, baseline kidney function testing, and confirmation that the patient will not use Viltepso with any other exon skipping agents.

Affected codes: J1427
Confirmed diagnosis of DMD
Genetic mutation amenable to exon 53 skipping
Age 4-9 years
Active corticosteroid use
Documentation of baseline kidney function testing prior to starting therapy
No concurrent use of other exon skipping agents (eg, Vyondys 53)

Documentation Required

Baseline and ongoing kidney monitoring

Obtain recommended baseline kidney tests before initiating Viltepso and monitor regularly during therapy due to observed kidney toxicity in animal studies and the potential unreliability of serum creatinine in DMD. Baseline testing should include serum cystatin C, urine dipstick, and urine protein-to-creatinine ratio. During treatment, perform urine dipstick monthly, and measure serum cystatin C and urine protein-to-creatinine ratio every 3 months. Refer the patient to a pediatric nephrologist for persistent elevations in serum cystatin C or for persistent proteinuria.

Baseline: serum cystatin C; urine dipstick; urine protein-to-creatinine ratio
Monthly: urine dipstick
Every 3 months: serum cystatin C and urine protein-to-creatinine ratio
Refer to pediatric nephrology for persistent abnormalities (elevated cystatin C or proteinuria)

Billing Rule

Quantity limit / dosing

Bill Viltepso using HCPCS J1427 (1 unit = 10 mg). Expected dosing is 80 mg/kg administered once weekly. Approval duration for coverage is 6 months.

Affected code: J1427
1 unit = 10 mg (HCPCS J1427)
Expected dosing: 80 mg/kg once weekly
Approval duration: 6 months

Background & Evidence

Viltepso (viltolarsen) is a phosphorodiamidate morpholino oligomer antisense oligonucleotide approved under accelerated approval to increase dystrophin production in skeletal muscle for DMD patients amenable to exon 53 skipping. It was studied in a phase II, multi-center, randomized, placebo-controlled, dose-finding trial in ambulatory boys ages 4-9 (Clemens 2020, NCT02740972) with functional secondary outcomes (6MWT, TTSTAND, TTRW, TTCLIMB, NSAA); a phase 3 trial (RACER53, NCT04060199) is ongoing. Although kidney toxicity was not observed in clinical studies, kidney toxicity was seen in animals who received viltolarsen, prompting recommended baseline and ongoing kidney monitoring.

Label	Summary
Phase 2 randomized placebo-controlled study	Clemens 2020 (NCT02740972) in ambulant boys ages 4-9; assessed dystrophin production and functional measures (secondary outcomes: 6MWT, TTCLIMB, TTRW, TTSTAND, NSAA).
Regulatory status	Viltepso approved under accelerated approval based on increased dystrophin production in skeletal muscle; continued approval may require confirmatory trial (Viltepso label).

Definition: Exon 53 skipping — a molecular approach applicable to certain DMD deletions (e.g., exons 45-52, 47-52, 48-52, 49-52, 50-52 and 52) that enables production of a shorter but partially functional dystrophin protein.

Revision History

02/25/2022document_created

Document created (New York specific policy created based on guidance released from NYSDOH); effective 04/01/2022. (Material change)

04/01/2022effective_dateLatest

Policy effective date 04/01/2022.

08/20/2021annual_review

Annual Review: No changes.

08/01/2021administrative_update

Administrative update to add documentation.

08/21/2020clinical_criteria_added

New clinical criteria and new quantity limit added for Viltepso; HCPCS and ICD-10 coding updates tracked (J1427 effective 04/01/2021). (Material change)