CurrentMass General Brigham Health PlanPolicy N/A

Exondys (eteplirsen) coverage for Duchenne muscular dystrophy

Policy governs prior authorization, quantity limits, and continuation criteria for Exondys 51 (eteplirsen) for patients with Duchenne muscular dystrophy whose DMD gene mutation is amenable to exon 51 skipping; applies to members under the specified Mass General Brigham Health Plan pharmacy benefit.

Policy Summary

PayerMass General Brigham Health Plan

PolicyExondys (eteplirsen) coverage for Duchenne muscular dystrophy

Policy CodePolicy N/A

Change TypeNo material changes

Effective DateJan 1, 2024

Next Review DateN/A

Key ActionObtain prior authorization and submit genetic testing documentation confirming a DMD exon 51–amenable mutation.

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

30max dose (mg/kg)

1806MWT min (m)

6 moinitial approval

12 moreauthorization

<14age limit

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Coverage Criteria for Exondys (eteplirsen)

Initial Therapy — Covered when ALL of the following are met

Covered when ALL of the following are met

Initial Therapy criteria: Member has a diagnosis of Duchenne Muscular Dystrophy (DMD).

Initial Therapy criteria: Genetic testing confirms DMD and documentation demonstrates a DMD gene mutation amenable to exon 51 skipping (submit supporting documentation; see Appendix for examples).

Initial Therapy criteria: Treatment with Exondys 51 is initiated before age 14.

Initial Therapy criteria: Member can achieve an average distance of at least 180 meters on the 6-minute walk test while walking independently.>= 180 meters

Initial Therapy criteria: Requested dose will not exceed 30 mg/kg.<= 30 mg/kg

Initial Therapy criteria: Medication is prescribed by or in consultation with a physician who specializes in treatment of DMD.

Initial approvals will be granted for 6 months (Limitations).

Continuation Therapy — Reauthorization (Continuation of Therapy) when ALL of the following are met

Reauthorization (Continuation of Therapy) when ALL of the following are met

Continuation criteria: Member has demonstrated documented response to therapy as evidenced by remaining ambulatory (e.g., able to walk with or without assistance and not wheelchair dependent).

Continuation criteria: Requested dose will not exceed 30 mg/kg.<= 30 mg/kg

Reauthorizations will be granted for 12 months (Limitations).

Not Medically Necessary

Not Medically Necessary: Use of Exondys 51 for indications other than Duchenne muscular dystrophy with a confirmed DMD gene mutation amenable to exon 51 skipping.

All other indications are considered experimental/investigational and not medically necessary.

All indications other than Duchenne muscular dystrophy (DMD) with a confirmed DMD gene mutation amenable to exon 51 skipping are considered experimental/investigational and not medically necessary. This policy does not cover Exondys 51 for diagnoses or uses outside the FDA‑approved indication.

Use of Exondys 51 (eteplirsen) for DMD patients whose mutation is not amenable to exon 51 skipping, or for other diseases or indications, is not medically necessary and will not be authorized under this policy.

Initial Authorization Criteria

Initial Therapy — Initial authorization criteria

Initial authorization criteria

Initial therapy nodes: Confirmed diagnosis of Duchenne muscular dystrophy (DMD).

Initial therapy nodes: Genetic testing documentation showing a DMD gene mutation amenable to exon 51 skipping (see Appendix examples such as deletion of exon 50, exon 52, or multi-exon deletions including 45-50, 47-50, 48-50, 49-50).

Initial therapy nodes: Initiation of treatment before age 14.< 14 years

Initial therapy nodes: 6-minute walk test result >= 180 meters while walking independently.>= 180 meters

Initial therapy nodes: Requested dose does not exceed 30 mg/kg (weight-based dosing).<= 30 mg/kg

Initial approval for 6 months.

Initial therapy nodes: Prescribed by or in consultation with a physician who specializes in treatment of DMD.

Provider Actions and Requirements

Denial Risk

Indication requirement

Use for indications other than Duchenne muscular dystrophy (DMD) with a confirmed DMD gene mutation that is amenable to exon 51 skipping is considered experimental / investigational and not medically necessary.

Prior Authorization

Prior authorization required

Prior authorization required for Exondys (eteplirsen) under the pharmacy/specialty benefit. Authorization may be granted for members new to the plan who are currently receiving treatment with the requested medication (excluding samples or manufacturer patient assistance program supplies).

Contact (All Plans) Phone: 800-711-4555 Fax: 844-403-1029
Specialty/All Plans Phone: 877-519-1908 Fax: 855-540-3693

Step Therapy

Step therapy

No specific step therapy alternatives are specified for Exondys in this document.

Documentation Required

Genetic testing and mutation documentation

Submit documentation confirming DMD diagnosis and genetic testing demonstrating a DMD gene mutation amenable to exon 51 skipping (examples in Appendix: deletions of exon 50, exon 52, exons 45–50, 47–50, 48–50, 49–50). Additional required documentation: confirmation that treatment was initiated before age 14, baseline 6-minute walk distance ≥ 180 meters, prescribed dose will not exceed 30 mg/kg, and that the prescriber is a physician who specializes in treatment of DMD.

Definitions

Exon 51–amenable mutation — examples and explanation

DefinitionA DMD gene mutation that is amenable to exon 51 skipping as determined by genetic testing

Appendix examplesDeletion of exon 50; deletion of exon 52; deletions of exons 45-50, 47-50, 48-50, and 49-50

Required documentationSubmission of documents supporting that the DMD gene mutation is amenable to exon 51 skipping (see Appendix examples)

Clinical implicationOnly patients with an exon 51–amenable mutation are eligible for Exondys 51 under this policy; other indications are experimental/investigational