CurrentNeighborhood Health Plan of Rhode IslandPolicy N/A

Exondys 51 (eteplirsen) coverage

Policy governs coverage criteria, dosing, and authorization requirements for Exondys 51 (eteplirsen) for members (Medicaid, Commercial, Medicare) of Neighborhood Health Plan of Rhode Island, including initial and renewal criteria for Duchenne muscular dystrophy patients amenable to exon 51 skipping.

Policy Summary

PayerNeighborhood Health Plan of Rhode Island

PolicyExondys 51 (eteplirsen) coverage

Policy CodePolicy N/A

Change TypeNo material change

Effective DateDec 1, 2019

Next Review DateN/A

Key ActionObtain prior authorization and provide baseline documentation of dystrophin level or timed function tests before initial approval.

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

1400max billable units/28d

30 mg/kg wklydose

6 moinitial auth

G71.01covered dx

Coverage Criteria for Exondys 51 (eteplirsen)

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Universal and indication-specific criteria: 1) Patient has a confirmed DMD gene mutation that is amenable to exon 51 skipping; 2) Patient is not on concomitant therapy with other DMD-directed antisense oligonucleotides (e.g., golodirsen, casimersen, viltolarsen); 3) Patient has never received and will not receive Elevidys (delandistrogene moxeparvovec-rokl) within 6 months of this request; 4) Patient has been on a stable dose of corticosteroids for at least 6 months unless contraindicated or intolerant; 5) Patient retains meaningful voluntary motor function (for example: able to speak, manipulate objects with upper extremities, ambulate, etc.); 6) Patient is receiving physical and/or occupational therapy; 7) Baseline documentation must be provided: dystrophin level OR 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 upper limb function (ULM) test OR North Star Ambulatory Assessment (NSAA) score OR Forced Vital Capacity (FVC) percent predicted.

Medicare members who received this medication within the past 365 days are exempt from Step Therapy requirements.

Renewal Therapy

Covered when ALL of the following are met at renewal

Renewal requirements: 1) Patient continues to meet universal and other indication-specific relevant criteria identified in Initial Approval (including concomitant therapy restrictions); 2) There is absence of unacceptable toxicity from the drug (examples include hypersensitivity reactions such as bronchospasm, chest pain, cough, tachycardia, urticaria); 3) Patient has demonstrated response to therapy compared to pretreatment baseline as evidenced by one or more of: increase in dystrophin level OR improvement in quality of life OR stability, improvement, or slowed rate of decline on timed function tests (6MWT, TTSTAND, TTRW, TTCLIMB) OR upper limb function (ULM) OR NSAA score OR FVC percent predicted.

Concomitant therapy with other DMD-directed antisense oligonucleotides is not permitted. Specifically, patients receiving Exondys 51 (eteplirsen) must not be on contemporaneous treatment with agents such as Vyondys 53 (golodirsen), Amondys 45 (casimersen), Viltepso (viltolarsen), or other DMD-targeted antisense oligonucleotides.

Use of Exondys 51 is not supported for patients who do not have a confirmed DMD gene mutation that is amenable to exon 51 skipping. In addition, requests lacking required baseline documentation are not permitted; acceptable baseline evidence includes at least one of the following: dystrophin level, timed function tests (for example, 6-minute walk test, TTSTAND, TTRW, TTCLIMB), upper limb function (ULM) test, North Star Ambulatory Assessment (NSAA) score, or forced vital capacity (FVC) percent predicted.

Coding, Units, and Diagnosis Codes

HCPCS CodeHCPCSCovered

J1428

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

NDCNDC

60923-0363-xx	Exondys 51 100 mg/2 mL single-dose vial
60923-0284-xx	Exondys 51 500 mg/10 mL single-dose vial

Covered Diagnosis CodesICD-10Covered

G71.01

Duchenne or Becker muscular dystrophy

inv-08: Max HCPCS units per period

Maximum billable units1400 billable units per 28 days

HCPCS unit definitionBilling uses HCPCS units; refer to HCPCS code J1428 for eteplirsen dosing (10 mg = 1 billable unit)

Billing periodLimit applies to each consecutive 28-day period

Provider Actions and Authorization Requirements

Prior Authorization

Prior Authorization Required

Prior authorization is required. Initial coverage will be provided only when all universal and DMD-specific criteria are met.

Prior authorization required for initial and renewal requests
Medicare members who received this medication within the past 365 days are exempt from step therapy requirements

Step Therapy

Step Therapy / Sequencing

Medicare members who have previously received this medication within the past 365 days are not subject to step therapy requirements. Patients on concomitant DMD-directed antisense oligonucleotide therapy (e.g., golodirsen, casimersen, viltolarsen) or who have received Elevidys within 6 months of this request are not eligible.

Medicare exemption: prior receipt within 365 days
Disallowed concomitant therapies: DMD-directed antisense oligonucleotides
Elevidys (delandistrogene moxeparvovec-rokl) within 6 months = exclusion

Documentation Required

Required Clinical Documentation

Baseline and supporting clinical documentation must be submitted with the prior authorization request to demonstrate eligibility.

Documented confirmed DMD gene mutation amenable to exon 51 skipping
Evidence of stable corticosteroid use for ≥ 6 months unless contraindicated or intolerant
Documentation of meaningful voluntary motor function and receipt of physical/occupational therapy
Baseline measurement of one or more: dystrophin level; timed function tests (6MWT, TTSTAND, TTRW, TTCLIMB); upper limb function (ULM) test; North Star Ambulatory Assessment (NSAA) score; Forced Vital Capacity (FVC) % predicted

Denial Risk

Triggers for Denial

Requests may be denied if the patient is on excluded concomitant therapy, has received Elevidys within the prior 6 months, or fails to meet the universal and DMD-specific criteria required for approval.

Concomitant DMD-directed antisense oligonucleotide therapy present = denial risk
Elevidys within 6 months = denial risk
Failure to provide required baseline documentation or to meet mutation, functional, or corticosteroid stability criteria = denial risk

Background

Exondys 51 (eteplirsen) is an antisense oligonucleotide indicated for the treatment of Duchenne muscular dystrophy in patients with a confirmed DMD gene mutation amenable to exon 51 skipping. Clinical use requires that patients meet universal criteria such as not being on concomitant DMD-directed antisense oligonucleotide therapy and other indication-specific requirements (for example, stability on corticosteroids when tolerated, meaningful voluntary motor function, and enrollment in physical or occupational therapy). Clinical trials have demonstrated increases in dystrophin in skeletal muscle; common adverse events reported include balance disorder and vomiting.

Definitions

inv-14: DMD amenable to exon 51 skipping

DefinitionA confirmed mutation of the DMD gene that is specifically responsive to exon 51 skipping therapy as documented by genetic testing.

Clinical implicationOnly patients with this mutation meet the FDA‑labeled indication for Exondys 51 (eteplirsen) and are eligible under the policy.

Documentation requiredGenetic testing results demonstrating the DMD mutation amenable to exon 51 skipping must be provided as baseline documentation.

Related evidenceClinical trials showed increased dystrophin in skeletal muscle for patients with DMD mutations amenable to exon 51 skipping.