CurrentNeighborhood Health Plan of Rhode IslandPolicy N/A

Vyondys 53 (golodirsen) intravenous therapy for Duchenne muscular dystrophy

Policy governs prior authorization, coverage criteria, dosing, renewal, allowable units, and billing codes for Vyondys 53 (golodirsen) for Medicaid, Commercial, and Medicare members of Neighborhood Health Plan of Rhode Island. Applies to outpatient intravenous administration with specific clinical and monitoring requirements.

Policy Summary

PayerNeighborhood Health Plan of Rhode Island

PolicyVyondys 53 (golodirsen) intravenous therapy for Duchenne muscular dystrophy

Policy CodePolicy N/A

Change TypeClarified

Effective DateMar 6, 2020

Next Review Date

Key ActionSubmit prior authorization with documentation of genetic confirmation amenable to exon 53 skipping, baseline dystrophin or timed function tests/ULM/NSAA/FVC, prior therapy history, and monitoring labs.

POLICY UPDATE CHANGES

Medicare members who previously received the medication within the past 365 days are not subject to step therapy requirements.

1Covered Diagnosis Codes

1400Maximum Units (per 28 days)

6 moAuthorization Length

Coverage Summary

Vyondys 53 (golodirsen) is indicated for treatment of Duchenne muscular dystrophy in patients with a confirmed DMD gene mutation amenable to exon 53 skipping, consistent with FDA-approved labeling. Clinical studies demonstrate an increase in dystrophin production in muscle tissue, and the policy requires baseline and periodic laboratory and clinical monitoring per labeling and supplemental plan requirements to mitigate toxicity risk. Coverage is provided with specified prior authorization criteria and renewals; the policy applies to Medicaid, Commercial, and Medicare members and governs outpatient intravenous administration, dosing limits (e.g., 30 mg/kg IV once weekly) and billing/units (e.g., 1400 billable units every 28 days), and site-of-care requirements as set by the plan.

Initial Approval Criteria

Coverage is provided when ALL of the following are met:

ALL of the following

Patient has a confirmed mutation of the DMD gene that is amenable to exon 53 skipping
Patient has been on a stable dose of corticosteroids for at least months, unless contraindicated or intolerant
Exact duration in months is incomplete/unspecified in source text
Patient retains meaningful voluntary motor function (e.g., able to speak, manipulate objects using upper extremities, ambulate)
Patient is receiving physical and/or occupational therapy
Baseline documentation requires ONE or more of the following
- 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] or 4-stair climb [4SC])
- Upper limb function (ULM) test
- North Star Ambulatory Assessment (NSAA) score
- Forced Vital Capacity (FVC) percent predicted
Patient had an inadequate response, or has a contraindication or intolerance, to Viltolarsen (Vitepso)
Patient is not on concomitant other DMD-directed antisense oligonucleotides (examples listed: eteplirsen/Exondys 51, casimersen/Amondys 45, viltolarsen/Viltepso, etc.)
Patient has never received and will not receive Elevidys (delandistrogene moxeparvovec-rokl) within 6 months of this request
Baseline laboratory monitoring: serum cystatin C, urine dipstick, and urine protein-to-creatinine ratio (UPCR) are measured prior to starting therapy and periodically during treatment

Continuation / Renewal Criteria

Renewal Criteria

For authorization renewal (every 6 months) ALL of the following must be met:

ALL of the following

Patient continues to meet universal and other indication-specific relevant criteria (including concomitant therapy requirements, performance status)
Absence of unacceptable toxicity
Examples of unacceptable toxicity include severe hypersensitivity reactions, kidney toxicity (e.g., glomerulonephritis, persistent increase in serum cystatin C, proteinuria)
Evidence of clinical benefit documented compared to pretreatment baseline (one or more required)
- Increase in dystrophin level
- Improvement in quality of life
- Stability, improvement, or slowed rate of decline in one or more of the following: timed function tests (6MWT, TTSTAND, TTRW, TTCLIMB/4SC), Upper limb function (ULM) test, NSAA score, FVC percent predicted

Length of Authorization

Authorization is valid for 6 months and may be renewed

Provider Actions & Prior Authorization

Prior Authorization

Prior authorization required

Authorization is required prior to dispensing. Initial approvals are valid for 6 months and may be renewed. Prior authorization review requires submission of documentation demonstrating genetic confirmation of a DMD mutation amenable to exon 53 skipping, baseline measures (dystrophin level and/or baseline timed function tests/ULM/NSAA/FVC), prior therapies (e.g., prior viltolarsen use or contraindications), and monitoring laboratory results (serum cystatin C, urine dipstick, UPCR).

Documentation Required

Baseline and ongoing monitoring documentation

Document baseline dystrophin level and/or baseline timed function tests (examples: 6-minute walk test, time to stand [TTSTAND], time to run/walk 10 meters [TTRW], time to climb 4 stairs [TTCLIMB] or 4-stair climb [4SC]), upper limb function (ULM) test, North Star Ambulatory Assessment (NSAA) score, and forced vital capacity (FVC) percent predicted. Measure serum cystatin C, urine dipstick, and urine protein-to-creatinine ratio (UPCR) prior to initiation and periodically during treatment; submit periodic monitoring results with renewal requests every 6 months.

Dystrophin level
Timed function tests (6MWT, TTSTAND, TTRW, TTCLIMB/4SC)
Upper limb function (ULM) test
NSAA score
FVC percent predicted
Serum cystatin C, urine dipstick, UPCR measured at baseline and periodically

Denial Risk

Concomitant therapy exclusions

Requests may be denied if the patient is receiving other DMD-directed antisense oligonucleotides (examples: eteplirsen/Exondys 51, casimersen/Amondys 45, viltolarsen/Viltepso) or if the patient has received or will receive Elevidys (delandistrogene moxeparvovec-rokl) within 6 months of the request.

Billing Rule

Unit billing equivalence

Bill using HCPCS code J1429 (Injection, golodirsen, 10 mg; 1 billable unit = 10 mg). Do not exceed 1400 billable units every 28 days. Note: this billing cap aligns with the dosing threshold of 30 mg/kg administered intravenously once weekly and the policy's maximum units per 28 days.

Background & Clinical Evidence

Vyondys 53 (golodirsen) is indicated for patients with Duchenne muscular dystrophy who have a DMD mutation amenable to exon 53 skipping. Clinical studies report golodirsen increases dystrophin production in muscle tissue — reflecting its exon-skipping mechanism that permits production of a truncated but partially functional dystrophin protein. Common adverse events observed include injection-site reactions, vomiting, and rash. The plan’s coverage criteria align with FDA labeling and the clinical literature and require supplemental baseline and periodic monitoring (including serum cystatin C, urine dipstick, and urine protein-to-creatinine ratio) and other documentation to mitigate and detect potential toxicity risks during therapy.

Primary regulatory source: Vyondys 53 package insert (Sarepta Therapeutics), June 2024 is cited as the plan’s primary regulatory reference supporting indication, dosing, and safety information.

Clinical trial finding: Published clinical reports and trials indicate golodirsen induces exon skipping that leads to sarcolemmal dystrophin expression in patients with mutations amenable to exon 53 skipping, demonstrating increased dystrophin production compared to baseline.

Max units per 28 days1400 billable units every 28 days

Dose30 mg/kg intravenously once weekly

Definitions

Meaningful voluntary motor function: Patient retains the ability to perform voluntary motor activities such as speaking, manipulating objects with the upper extremities, and ambulating (examples provided in policy as criteria for therapy initiation).

Unacceptable toxicity: Includes severe hypersensitivity reactions and kidney toxicity examples such as glomerulonephritis, persistent increase in serum cystatin C, and proteinuria; these adverse events are grounds for treatment discontinuation or denial on renewal.