CurrentMass General Brigham Health PlanPolicy N/A

Vyondys 53 (golodirsen) — Coverage Criteria

Policy governing prior authorization, reauthorization, and coverage criteria for golodirsen (Vyondys 53) for members with Duchenne muscular dystrophy whose DMD mutation is amenable to exon 53 skipping; applies to Mass General Brigham Health Plan members.

Policy Summary

PayerMass General Brigham Health Plan

PolicyVyondys 53 (golodirsen) — Coverage Criteria

Policy CodePolicy N/A

Change TypeNo material clinical or coverage changes

Effective DateDec 1, 2020

Next Review DateN/A

Key ActionObtain prior authorization with documentation confirming DMD diagnosis and a mutation amenable to exon 53 skipping; prescribing physician must be a neurologist or DMD specialist.

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

6 monthsInitial approval

12 monthsReauth interval

exon 53Target mutation

neurologistPrescribing specialty

Coverage Criteria for Vyondys 53 (golodirsen)

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Initial Therapy

Covered when ALL of the following are met

Initial Authorization: 1) Prescribing physician is a neurologist or a provider who specializes in the treatment of DMD; 2) Member has a diagnosis of Duchenne muscular dystrophy with medical records confirming a DMD mutation amenable to exon 53 skipping; 3) Baseline 6-minute walk test (6MWT) documented with distance ≥ 200 meters (test observed or completed by the treating provider, or ordered by the treating provider and completed by a qualified medical practitioner); 4) Member has been receiving a stable dose of corticosteroids for at least 6 months OR has a documented contraindication to corticosteroids; 5) Member is not on concomitant therapy with other DMD-directed antisense oligonucleotides.6MWT >= 200 meters

6MWT must be documented and observed or completed as specified; corticosteroid stability defined as >= 6 months.

Continuation Therapy

Reauthorizations may be approved for 12 months when ALL of the following are met

Reauthorization: 1) Member remains ambulatory as defined by a current 6-minute walk test (6MWT) ≥ 200 meters (test observed or completed by the treating provider, or ordered by the treating provider and completed by a qualified medical practitioner); 2) Member demonstrates a stable or improving pattern of 6MWTs with medical records that include a pretreatment baseline and all interim results (all previous 6MWT results must be provided); 3) Member continues corticosteroid therapy in combination with the requested agent OR has a documented contraindication to corticosteroids.6MWT >= 200 meters

All prior 6MWT results (baseline and interim) must be included in the medical record for reauthorization review.

Coding and Measurement Requirements

Pharmacy benefit / Prior authorization contactmixed

No codes listed

6-Minute Walk Test (6MWT) — ambulatory criteria and reauthorization thresholds

Ambulatory definition (6MWT)Member is ambulatory as defined by a current 6-minute walk test (6MWT) ≥ 200 meters (test observed/completed by treating provider or ordered and completed by a qualified practitioner).

Baseline requirement (initial)Baseline 6MWT must be documented and observed/completed as specified prior to initiation of therapy.

Reauthorization stabilityReauthorization requires a stable or improving pattern of 6MWTs with pretreatment baseline and all interim results provided (all previous 6MWT results must be included).

Reauthorization intervalWhen criteria are met, reauthorizations may be approved for 12 months.

Test administration6MWT must be observed or completed by the treating provider, or ordered by the treating provider and completed by a qualified medical practitioner.

Provider Requirements and Actions

Prior Authorization

Prior Authorization Required — Prescribing Physician Specialty

Prior authorization required — submit prior auth to Mass General Brigham Health Plan for Vyondys 53 (golodirsen). The prescribing physician must be a neurologist or a provider who specializes in the treatment of Duchenne muscular dystrophy (DMD).

Contact (All Plans): Phone: 877-519-1908; Fax: 855-540-3693
Benefit/Program: Pharmacy Benefit, Quantity Limit, Prior Authorization
Specialty Limitations: N/A

Step Therapy

Step Therapy — None Specified

No step therapy or specialty step restrictions listed for Vyondys 53 in the source policy. Do not apply step therapy requirements beyond standard prior authorization and reauthorization criteria.

Documentation Required

Required Supporting Records

Provide supporting documentation with the request: medical records confirming diagnosis and DMD mutation amenable to exon 53 skipping; baseline and interim 6-minute walk test (6MWT) results where applicable; documentation of corticosteroid use or contraindication; and records confirming member is not on other DMD-directed antisense oligonucleotides.

Medical records confirming DMD diagnosis and mutation amenable to exon 53 skipping
Baseline and interim 6-minute walk test (6MWT) results (observed/completed by treating provider or completed by qualified practitioner)
Documentation of stable corticosteroid use for ≥6 months OR documentation of contraindication to corticosteroids
Documentation confirming member is not receiving other DMD-directed antisense oligonucleotide therapy

Denial Risk

Documentation and Current Therapy — Denial Risk if Not Currently Receiving Vyondys 53

Reauthorization: Authorization may be granted for members who are currently receiving treatment with Vyondys 53; exclusions apply if product was obtained only as samples or via manufacturer patient assistance program. Failure to document that the member is currently receiving Vyondys 53 (not limited to samples or patient assistance) or to submit required records may result in denial.

Member must be currently receiving Vyondys 53 (excludes samples or manufacturer patient assistance program)
Meet all reauthorization criteria per policy

Definitions

Amenable to exon 53 skipping — definition of mutation amenability

DefinitionA DMD gene mutation that is amenable to exon 53 skipping (i.e., a confirmed mutation of the DMD gene for which exon 53 skipping therapy is appropriate).

Documentation requiredMedical records must confirm the member has DMD with a mutation of the DMD gene that is amenable to exon 53 skipping.

Therapy specificityGolodirsen (Vyondys 53) is indicated only for patients with DMD mutations amenable to exon 53 skipping; eligibility hinges on this molecular characterization.