CurrentNew Hampshire Department of Health and Human ServicesPolicy N/A

Duchenne Muscular Dystrophy (DMD) Agents Criteria

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Prior authorization and renewal coverage for eteplirsen, viltolarsen, golodirsen, and casimersen for Duchenne muscular dystrophy under the New Hampshire Medicaid Fee-for-Service Program.

Policy Summary

PayerNew Hampshire Department of Health and Human Services

PolicyDuchenne Muscular Dystrophy (DMD) Agents Criteria

Policy CodePolicy N/A

Change TypeNo material change

Effective DateN/A

Next Review DateN/A

Key ActionSubmit documentation of the confirmed DMD diagnosis, applicable mutation, clinical eligibility, baseline measures, and agent-specific safety monitoring for prior authorization.

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

4DMD agents covered by the criteria

3Exon-skipping targets addressed

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CurrentNew Hampshire Department of Health and Human ServicesPolicy N/A

Duchenne Muscular Dystrophy (DMD) Agents Criteria

Customize your policy alerts

Sign up for all New Hampshire Department of Health and Human Services policy alerts

Know when New Hampshire Department of Health and Human Services releases new policies or updates existing guidance.

Monitor payer policy activity

Receive email alerts as soon as relevant payer policies are published or updated.

Prior authorization and renewal coverage for eteplirsen, viltolarsen, golodirsen, and casimersen for Duchenne muscular dystrophy under the New Hampshire Medicaid Fee-for-Service Program.

Policy Summary

PayerNew Hampshire Department of Health and Human Services

PolicyDuchenne Muscular Dystrophy (DMD) Agents Criteria

Policy CodePolicy N/A

Change TypeNo material change

Effective DateN/A

Next Review DateN/A

Key ActionSubmit documentation of the confirmed DMD diagnosis, applicable mutation, clinical eligibility, baseline measures, and agent-specific safety monitoring for prior authorization.

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

4DMD agents covered by the criteria

3Exon-skipping targets addressed

6 monthsInitial authorization period

6 monthsRenewal authorization period

Monthly / every 3 monthsMonitoring schedule for selected agents

Coverage Criteria

Initial Therapy Criteria

Covered when all applicable criteria are met.

Initial therapy

Diagnosis and mutation: Confirmed diagnosis of Duchenne muscular dystrophy with genetic testing demonstrating a mutation on the DMD gene amenable to the applicable exon-skipping target: exon 45 for Amondys 45, exon 51 for Exondys 51, or exon 53 for Viltepso or Vyondys 53.
Stable corticosteroid therapy
- Six-month corticosteroid duration: Stable corticosteroid dose for at least 6 months for Amondys 45, Exondys 51, or Vyondys 53.≥6 months
- Three-month corticosteroid duration: Stable corticosteroid dose for at least 3 months for Viltepso.≥3 months
Motor function: Patient retains meaningful voluntary motor function, such as the ability to speak, manipulate objects using the upper extremities, or ambulate.
Rehabilitation therapy: Patient should be receiving physical therapy and/or occupational therapy.
Baseline assessment: Baseline documentation includes at least one of the following: dystrophin level; 6-minute walk test or other timed function test; upper limb function test; North Star Ambulatory Assessment; or FVC percentage predicted.≥1 measure
Agent-specific renal monitoring: For Amondys 45, Vyondys 53, and Viltepso, serum cystatin C, urine dipstick, and urine protein-to-creatinine ratio are measured before treatment, with prescriber attestation that monitoring will continue during treatment.Before treatment; monthly urine dipstick; serum cystatin C and urine protein-to-creatinine ratio every 3 months
Applies only to Amondys 45, Vyondys 53, and Viltepso.
Viltepso cardiac safety: For Viltepso, patient does not have symptomatic cardiomyopathy.
Applies only to Viltepso.

Continuation Therapy Criteria

Covered for a 6-month renewal when all applicable criteria are met.

Six-month renewal

Continued eligibility: Patient continues to meet the initial therapy criteria.
Response to therapy
- Dystrophin response: Increase in dystrophin level.Increase
- Timed function response: Stability, improvement, or slowed rate of decline in the 6-minute walk test or other timed function tests.Stable, improved, or slowed decline

Unacceptable toxicity from therapy is an exclusion to coverage.

Coverage for Viltepso is excluded when the patient has symptomatic cardiomyopathy.

Prior Authorization and Provider Requirements

Prior Authorization

Initial Prior Authorization

Submit an initial prior authorization request with documentation supporting the applicable DMD agent criteria. Initial authorization is limited to 6 months.

Document the confirmed DMD diagnosis, qualifying exon-skipping mutation, clinical eligibility, baseline assessment, and any agent-specific monitoring requirements.
Approval may be extended for 6 months only when the renewal criteria are met.

Prior Authorization

Six-Month Renewal Authorization

Submit a six-month renewal authorization request with documentation that the patient continues to meet the initial criteria and has demonstrated response without treatment-restricting adverse effects.

Renewal authorization is for an additional 6 months when the required response, safety, and continuation documentation is provided.

Policy Background

This policy addresses treatment of Duchenne muscular dystrophy (DMD) with antisense oligonucleotide therapies that promote skipping of a mutation-specific exon in the DMD gene. The covered agents target exon 45, exon 51, or exon 53: Amondys 45 targets exon 45, Exondys 51 targets exon 51, and Viltepso or Vyondys 53 target exon 53. Coverage also requires preserved meaningful voluntary motor function and supporting baseline and safety monitoring documentation.

Key Definitions

Mutation amenable to exon skipping

DefinitionA confirmed mutation of the DMD gene that can be treated by skipping the specific exon targeted by the agent.

Exon 45 targetFor Amondys 45 (casimersen), the mutation is amenable to exon 45 skipping.

Exon 51 targetFor Exondys 51 (eteplirsen), the mutation is amenable to exon 51 skipping.

Exon 53 targetFor Viltepso (viltolarsen) or Vyondys 53 (golodirsen), the mutation is amenable to exon 53 skipping.

Meaningful voluntary motor function

DefinitionRetained voluntary motor function demonstrated by the ability to speak, manipulate objects using the upper extremities, or ambulate.

Initial Approval Criteria

Initial Approval

Initial approval requires all applicable mutation-specific, clinical, baseline-testing, and safety-monitoring criteria.

Initial approval

Diagnosis and mutation: Confirmed diagnosis of Duchenne muscular dystrophy with genetic testing demonstrating a mutation on the DMD gene amenable to the applicable exon-skipping target: exon 45 for Amondys 45, exon 51 for Exondys 51, or exon 53 for Viltepso or Vyondys 53.
Stable corticosteroid therapy
- Six-month corticosteroid duration: Stable corticosteroid dose for at least 6 months for Amondys 45, Exondys 51, or Vyondys 53.≥6 months

Six-Month Renewal Criteria

Six-Month Renewal

Renewal authorization is available for 6 months when all continuation requirements are met.

Six-month renewal

Continued eligibility: Patient continues to meet the initial approval criteria.
Response to therapy
- Dystrophin response: Increase in dystrophin level.Increase
- Timed function response: Stability, improvement, or slowed rate of decline in the 6-minute walk test or other timed function tests.

Policy Summary

PayerNew Hampshire Department of Health and Human Services

PolicyDuchenne Muscular Dystrophy (DMD) Agents Criteria

Policy CodePolicy N/A

Change TypeNo material change

Effective DateN/A

Next Review DateN/A

Key ActionSubmit documentation of the confirmed DMD diagnosis, applicable mutation, clinical eligibility, baseline measures, and agent-specific safety monitoring for prior authorization.

SourceLink

On This Page

Upper limb response: Stability, improvement, or slowed rate of decline in the upper limb function test.Stable, improved, or slowed decline

NSAA response: Stability, improvement, or slowed rate of decline in the North Star Ambulatory Assessment.Stable, improved, or slowed decline

Respiratory response: Stability, improvement, or slowed rate of decline in FVC percentage predicted.Stable, improved, or slowed decline

Quality-of-life response: Improvement in quality of life.Improvement

Tolerability: Patient has not experienced treatment-restricting adverse effects, including severe hypersensitivity reactions or renal toxicity/proteinuria.

Documentation Required

Diagnosis and Mutation Documentation

Document a confirmed diagnosis of Duchenne muscular dystrophy with genetic testing identifying a mutation amenable to the applicable exon-skipping therapy.

Amondys 45: mutation amenable to exon 45 skipping.
Exondys 51: mutation amenable to exon 51 skipping.
Viltepso or Vyondys 53: mutation amenable to exon 53 skipping.

Documentation Required

Clinical Eligibility and Baseline Measures

Document clinical eligibility before initial approval, including corticosteroid treatment, meaningful voluntary motor function, supportive therapy, and baseline disease measures.

Stable corticosteroid dose unless contraindicated or intolerable: at least 6 months for Amondys 45, Exondys 51, or Vyondys 53; at least 3 months for Viltepso.
Patient retains meaningful voluntary motor function, such as the ability to speak, manipulate objects using the upper extremities, or ambulate.
Patient is receiving physical therapy and/or occupational therapy.
Provide at least one baseline measure: dystrophin level; 6-minute walk test or another timed function test; upper limb function test; North Star Ambulatory Assessment; or forced vital capacity percentage predicted.

Documentation Required

Renal Monitoring Documentation

For Amondys 45, Vyondys 53, and Viltepso, document required renal assessments before therapy and attest to continued monitoring during treatment.

Before therapy: measure serum cystatin C, urine dipstick, and urine protein-to-creatinine ratio.
During therapy: perform a monthly urine dipstick and measure serum cystatin C and urine protein-to-creatinine ratio every 3 months.

Documentation Required

Renewal Response and Safety Documentation

For renewal, document that the patient continues to meet the applicable criteria and has responded to therapy compared with the pretreatment baseline.

Document one or more of the following: increased dystrophin level; stability, improvement, or slowed decline in 6-minute walk or other timed function tests; stability, improvement, or slowed decline in upper limb function; stability, improvement, or slowed decline in NSAA; stability, improvement, or slowed decline in FVC percentage predicted; or improved quality of life.
Document that the patient has not experienced treatment-restricting adverse effects, including severe hypersensitivity reactions or renal toxicity/proteinuria.

Denial Risk

Criteria Not Met or Unacceptable Toxicity

The request may be denied when the applicable approval criteria are not met or when the patient has unacceptable toxicity from therapy.

Criteria not met.
Unacceptable toxicity from therapy.

Denial Risk

Failure to Respond or Treatment-Restricting Toxicity

Renewal may be denied if the patient fails to demonstrate a response compared with pretreatment baseline or develops treatment-restricting toxicity.

Failure to show improvement, stability, or slowed decline in an applicable response measure may prevent continuation.
Severe hypersensitivity reactions, renal toxicity/proteinuria, or other unacceptable treatment-restricting adverse effects may prevent renewal.

Denial Risk

Symptomatic Cardiomyopathy with Viltepso

Before requesting Viltepso, document that the patient does not have symptomatic cardiomyopathy.

Viltepso is not approvable when symptomatic cardiomyopathy is present.

SpeechThe patient can speak.

Upper-extremity functionThe patient can manipulate objects using the upper extremities.

AmbulationThe patient can ambulate.

6-minute walk test (6MWT)

DefinitionThe 6-minute walk test (6MWT) or another timed function test used to assess motor function.

Baseline useDocument the 6MWT or another timed function test at baseline, when available, as one of the accepted baseline measures.

Response useFor renewal, compare the 6MWT or other timed function test with the pretreatment baseline; stability, improvement, or a slowed rate of decline demonstrates response.

Three-month corticosteroid duration:

Stable corticosteroid dose for at least 3 months for Viltepso.

Meaningful voluntary motor function: Patient retains meaningful voluntary motor function, such as the ability to speak, manipulate objects using the upper extremities, or ambulate.

Physical or occupational therapy: Patient should be receiving physical therapy and/or occupational therapy.

Baseline functional or disease assessment: Baseline documentation includes at least one of the following: dystrophin level; 6-minute walk test or other timed function test; upper limb function test; North Star Ambulatory Assessment; or FVC percentage predicted.≥1 measure

Agent-specific renal monitoring: For Amondys 45, Vyondys 53, and Viltepso, serum cystatin C, urine dipstick, and urine protein-to-creatinine ratio are measured before treatment, with prescriber attestation that monitoring will continue during treatment.Before treatment; monthly urine dipstick; serum cystatin C and urine protein-to-creatinine ratio every 3 months

Applies only to Amondys 45, Vyondys 53, and Viltepso.

Viltepso cardiac safety: For Viltepso, patient does not have symptomatic cardiomyopathy.

Applies only to Viltepso.

Stable, improved, or slowed decline

Upper limb response: Stability, improvement, or slowed rate of decline in the upper limb function test.Stable, improved, or slowed decline

NSAA response: Stability, improvement, or slowed rate of decline in the North Star Ambulatory Assessment.Stable, improved, or slowed decline

Respiratory response: Stability, improvement, or slowed rate of decline in FVC percentage predicted.Stable, improved, or slowed decline

Quality-of-life response: Improvement in quality of life.Improvement

Treatment tolerability: Patient has not experienced treatment-restricting adverse effects, including severe hypersensitivity reactions or renal toxicity/proteinuria.