CurrentViva HealthPolicy IC-0520

Vyondys 53 (golodirsen) IV for Duchenne Muscular Dystrophy (exon 53 skipping)

Policy governing prior authorization, coverage criteria, dosing, coding, and renewal requirements for intravenous golodirsen (Vyondys 53) for members with Duchenne muscular dystrophy amenable to exon 53 skipping.

Policy Summary

PayerViva Health

PolicyVyondys 53 (golodirsen) IV for Duchenne Muscular Dystrophy (exon 53 skipping)

Policy CodePolicy IC-0520

Change TypeNo material change

Effective DateJan 6, 2020

Next Review Date

Key ActionPrior authorization is required; document baseline renal tests and dystrophin or functional measures and request initial authorization for 180 days with renewals every 365 days.

POLICY UPDATE CHANGES

No material clinical or coverage changes

1Covered Diagnoses

1400Max Dose Units (per 28 days)

6Initial Auth (months)

12Renewal Interval (months)

Coverage Summary

Scope: This policy governs prior authorization, coverage criteria, dosing, coding, and renewal requirements for intravenous golodirsen (Vyondys 53) for members with Duchenne muscular dystrophy who have DMD gene mutations amenable to exon 53 skipping. Subject: Vyondys 53 (golodirsen) IV for Duchenne Muscular Dystrophy (exon 53 skipping). Policy number: IC-0520. Coverage stance: covered_with_criteria. Brief summary: Golodirsen (Vyondys 53) is an antisense oligonucleotide administered intravenously for patients with Duchenne muscular dystrophy with mutations amenable to exon 53 skipping; coverage is provided with prior authorization when specified clinical and monitoring criteria are met (including baseline and periodic renal monitoring, documentation of baseline functional measures or dystrophin level, corticosteroid stability or documented contraindication/intolerance, and exclusion of concomitant DMD-directed antisense oligonucleotides or delandistrogene moxeparvovec-rokl). Maximum authorized dosing and coding limits apply.

Initial Therapy Criteria

Initial Approval Criteria

Prior authorization validity is provided when ALL of the following universal and DMD-specific criteria are met:

ALL of the following

Universal Criteria
- Member is not on concomitant therapy with other DMD-directed antisense oligonucleotides
- Member is not on concomitant therapy with delandistrogene moxeparvovec-rokl
- Serum cystatin C, urine dipstick, and urine protein-to-creatinine ratio (UPCR) are measured prior to starting therapy and periodically during treatment
Duchenne Muscular Dystrophy (DMD) specific
- Member has a confirmed mutation of the DMD gene that is amenable to exon 53 skipping
- Member has been on a stable dose of corticosteroids, unless there is a contraindication or intolerance, for at least 6 months
- Member retains meaningful voluntary motor function (e.g., member is able to speak, manipulate objects using upper extremities, ambulate, etc.)
- Member is receiving physical and/or occupational therapy
- Baseline documentation (one or more required)
  - Dystrophin level
  - 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.)
  - Upper limb function (ULM) test
  - North Star Ambulatory Assessment (NSAA) score
  - Forced Vital Capacity (FVC) percent predicted
- Member had an inadequate response, or has a contraindication or intolerance, to viltolarsen

Prior Authorization

Prior authorization required

Prior authorization is required. Initial authorization is provided for 180 days and renewals may be granted every 365 days. Requests must document the criteria listed in Initial Approval (e.g., mutation amenable to exon 53 skipping, baseline measures, corticosteroid stability or contraindication, functional status, prior viltolarsen response/intolerance).

Affected codes: J1429
Affected diagnosis code: G71.01

Documentation Required

Baseline and periodic laboratory monitoring

Obtain serum cystatin C, urine dipstick, and urine protein-to-creatinine ratio (UPCR) prior to starting therapy and periodically during treatment. Document baseline dystrophin and/or one or more baseline functional/dystrophin measures.

Required labs: serum cystatin C
Required labs: urine dipstick
Required labs: urine protein-to-creatinine ratio (UPCR)
Baseline functional/dystrophin measures (one or more): 6-minute walk test (6MWT)
Timed tests: time to stand (TTSTAND), time to run/walk 10 meters (TTRW), time to climb 4 stairs (TTCLIMB)
Upper limb function (ULM) test
North Star Ambulatory Assessment (NSAA) score
Forced Vital Capacity (FVC) percent predicted

Continuation (Renewal) Criteria

Renewal Criteria

Prior authorization may be renewed when ALL of the following are met:

ALL of the following

Member continues to meet the universal and other indication-specific relevant criteria identified in Initial Approval (section III), including concomitant therapy requirements and performance status
Absence of unacceptable toxicity from the drug (examples include: severe hypersensitivity reactions, kidney toxicity such as glomerulonephritis, persistent increase in serum cystatin C, proteinuria, etc.)
ALL of the following
- Increase in dystrophin level
- Improvement in quality of life
- Stability, improvement, or slowed rate of decline in timed function tests (e.g., 6MWT, TTSTAND, TTRW, TTCLIMB)
- Upper limb function (ULM) test
- North Star Ambulatory Assessment (NSAA) score
- Forced Vital Capacity (FVC) percent predicted

Response examples (one or more)

Denial Risk

Concomitant therapy exclusions

Claims may be denied if the member is receiving excluded concomitant therapies. Specifically, members on other DMD-directed antisense oligonucleotides or on delandistrogene moxeparvovec-rokl may have claims denied.

Affected code: J1429
Exclusion: other DMD-directed antisense oligonucleotides
Exclusion: delandistrogene moxeparvovec-rokl

Billing Rule

HCPCS unit billing

Report J1429 with units based on 10 mg per billable unit. Do not exceed the maximum of 1400 billable units in any 28-day period.

J1429 billing: 1 billable unit = 10 mg
Maximum: 1400 units per 28-day period

Applicable Codes

Covered HCPCSHCPCSCovered

J1429

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

Covered NDCNDCCovered

60923-0465-xx

Vyondys 53 100 mg/2 mL single-dose vial

Covered Diagnosis CodesICD-10Covered

G71.01

Duchenne or Becker muscular dystrophy

Billable unit: For HCPCS code J1429, 1 billable unit = 10 mg of golodirsen. Coding note: report J1429 with units based on 10 mg per unit and convert the total administered dose to units accordingly (e.g., a 30 mg/kg weekly dose should be converted to the equivalent number of 10 mg units for billing). Vial size/NDC: Vyondys 53 is supplied as a 100 mg/2 mL single-dose vial (NDC 60923-0465-xx), so multiple vials may be required to prepare the calculated dose; do not exceed the plan limit of 1400 billable units every 28 days.

Clinical Evidence & Background

Background: Golodirsen is an antisense oligonucleotide that induces exon 53 skipping to enable production of internally truncated dystrophin in patients with Duchenne muscular dystrophy whose mutations are amenable to exon 53 skipping. The FDA-approved indication is for DMD patients with mutations amenable to exon 53 skipping (see label). Therapy requires baseline and periodic renal monitoring (serum cystatin C, urine dipstick, and urine protein-to-creatinine ratio) due to potential kidney toxicity. Prior authorization requires documentation of baseline dystrophin level or functional measures (examples include 6-minute walk test, TTSTAND, TTRW, TTCLIMB, ULM, NSAA, FVC% predicted) and confirmation of mutation amenable to exon 53 skipping. Members should be on a stable dose of corticosteroids for at least 6 months unless contraindicated or intolerant. See the FDA label (Vyondys 53 package insert, June 2024) and selected publications (e.g., Neurology 2020; Nucleic Acid Ther. 2022) for clinical trial results, dosing, and safety details.

30 mg/kgDose: 30 mg/kg IV once weekly

1400Max 1400 billable units per 28 days (1 unit = 10 mg)

Definitions: Billable unit — For J1429, 1 billable unit = 10 mg of golodirsen. Amenable to exon 53 skipping — a confirmed DMD gene mutation predicted to respond to exon 53 skipping therapy.

Provider Actions & Documentation Requirements

Note

Actionable provider requirements (summary)

Actionable requirements for golodirsen (Vyondys 53):

Prior authorization (prior_auth): Prior authorization is required for J1429 for members with DMD (G71.01). Initial approval provided for 180 days; renewals may be granted every 365 days. Documentation must support criteria in Initial Approval (e.g., mutation amenable to exon 53 skipping, corticosteroid status, functional status, prior viltolarsen response).
Documentation (documentation): Obtain serum cystatin C, urine dipstick, and UPCR prior to starting therapy and periodically during treatment. Document baseline dystrophin or one or more functional/dystrophin measures (6MWT, TTSTAND, TTRW, TTCLIMB, ULM, NSAA, FVC%).
Denial risk (denial_risk): Claims for J1429 may be denied if the member is on other DMD-directed antisense oligonucleotides or on delandistrogene moxeparvovec-rokl.
Billing rule (billing_rule): For J1429 report 1 billable unit = 10 mg; do not exceed 1400 units per 28 days.