CurrentWellcare HawaiiPolicy CP.PHAR.453

Clinical Policy: Golodirsen (Vyondys 53)

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This policy governs prior authorization and medical-necessity coverage of intravenous golodirsen for members with Duchenne muscular dystrophy caused by a DMD mutation amenable to exon 53 skipping. It applies to commercial, health insurance marketplace, and Medicaid lines of business administered under the policy.

Policy Summary

PayerWellcare Hawaii

PolicyGolodirsen (Vyondys 53) coverage for Duchenne muscular dystrophy

Policy CodePolicy CP.PHAR.453

Change TypeRevised

Effective Date03/01/2020

Last ReviewDec 1, 2020

Next Review DateN/A

Key ActionSubmit prior authorization documentation showing that all applicable initial or continuation approval criteria are met.

SourceLink

POLICY UPDATE CHANGES

Continuation therapy criteria were expanded to allow coverage for members receiving golodirsen through another health insurer or responding positively with stable disease.

The assessment window for continuation-therapy response parameters was changed from within 30 days to within 6 months.

Continuation therapy now requires assessment by a neurologist within the preceding 6 months.

Examples of positive response were removed from Appendix D because the specific measures are required in the continuation criteria.

30 mg/kgMaximum golodirsen dose per week

6 monthsApproval duration

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CurrentWellcare HawaiiPolicy CP.PHAR.453

Clinical Policy: Golodirsen (Vyondys 53)

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Policy Summary

PayerWellcare Hawaii

PolicyGolodirsen (Vyondys 53) coverage for Duchenne muscular dystrophy

Policy CodePolicy CP.PHAR.453

Change TypeRevised

Effective Date03/01/2020

Last ReviewDec 1, 2020

Next Review DateN/A

Key ActionSubmit prior authorization documentation showing that all applicable initial or continuation approval criteria are met.

SourceLink

POLICY UPDATE CHANGES

Continuation therapy criteria were expanded to allow coverage for members receiving golodirsen through another health insurer or responding positively with stable disease.

The assessment window for continuation-therapy response parameters was changed from within 30 days to within 6 months.

Continuation therapy now requires assessment by a neurologist within the preceding 6 months.

Examples of positive response were removed from Appendix D because the specific measures are required in the continuation criteria.

30 mg/kgMaximum golodirsen dose per week

6 monthsApproval duration

≤15 yearsMaximum age at initiation

≥250 m6-minute walk test

Coverage Criteria

Initial Therapy

Covered when all of the following initial approval criteria are met.

Initial therapy

Disease and prescriber
- The member has Duchenne muscular dystrophy with a mutation amenable to exon 53 skipping, confirmed by genetic testing.
- Vyondys 53 is prescribed by or in consultation with a neurologist.
Age and baseline clinical status
- The member is 15 years of age or younger at therapy initiation.Age ≤15 years
- Within the last 30 days, the member has ambulatory function, including the ability to walk with or without assistive devices, is not wheelchair dependent, and has a 6-minute walk test distance of at least 250 meters.6MWT ≥250 m
- Within the last 30 days, the member has stable cardiac function with a left ventricular ejection fraction greater than 50%.LVEF >50%
- Within the last 30 days, the member has stable pulmonary function with predicted forced vital capacity of at least 50%.Predicted FVC ≥50%
Corticosteroid history and concurrent treatment
- The member has had an inadequate response, evidenced by a significant decline in 6MWT, LVEF, or FVC, despite adherent use of an oral corticosteroid for at least 6 months, unless the corticosteroid is contraindicated or clinically significant adverse effects are experienced.Oral corticosteroid use ≥6 months
- Vyondys 53 is prescribed concurrently with an oral corticosteroid, unless contraindicated or clinically significant adverse effects are experienced.
Therapy restrictions and dosing
- Vyondys 53 is not prescribed concurrently with another exon-skipping therapy, such as Exondys 51.
- The dose does not exceed 30 mg/kg per week.≤30 mg/kg per week

Continuation Therapy

Covered when all of the following continuation criteria are met.

Continuation therapy

The member is currently receiving medication for DMD with a mutation amenable to exon 53 skipping, or previously met the initial approval criteria.
One permitted positive-response pathway
- Standard objective response pathway
  - Within the last 6 months, the member has ambulatory function, including the ability to walk with or without assistive devices, is not wheelchair dependent, and has a 6-minute walk test distance of at least 250 meters.6MWT ≥250 m
  - Within the last 6 months, the member has stable cardiac function with a left ventricular ejection fraction greater than 50%.LVEF >50%

Concurrent exon-skipping therapy is excluded from coverage. Vyondys 53 must not be prescribed or administered concurrently with another exon-skipping therapy, such as Exondys 51, for either initial or continued therapy.

Unlisted diagnoses or indications are not authorized under this policy. For a diagnosis or indication not specifically listed, consult the applicable off-label use policy for the member’s line of business.

Clinical Thresholds and Dose Limits

6-Minute Walk Test

Required distanceAt least 250 meters

Clinical measureAmbulatory function assessed by the six-minute walk test

Assessment timingWithin the last 30 days for initial therapy and within the last 6 months for continuation therapy

Left Ventricular Ejection Fraction

Required valueAbove 50%

Clinical statusStable cardiac function

Assessment timingWithin the last 30 days for initial therapy and within the last 6 months for continuation therapy

Authorization and Documentation Requirements

Prior Authorization

Prior Authorization Required: Vyondys 53

Prior authorization is required for Vyondys 53 for both initial and continuation therapy. Submit the applicable clinical documentation demonstrating that all approval criteria are met; approved therapy duration is 6 months.

Initial requests must document the qualifying diagnosis, mutation, age, prescriber, clinical assessments, corticosteroid history, concurrent corticosteroid use, absence of another exon-skipping therapy, and dose within limits.
Continuation requests must document current or prior qualifying therapy, treatment response, neurologist assessment, concurrent corticosteroid use, absence of another exon-skipping therapy, and dose within limits.

Prior Authorization

Prior Authorization Required: Emflaza

Prior authorization is required for Emflaza (deflazacort) when it is used as the oral corticosteroid supporting Vyondys 53 approval. Document the corticosteroid therapy and any applicable contraindication or clinically significant adverse effect.

Initial approval generally requires inadequate response despite adherent oral corticosteroid use for at least 6 months, unless contraindicated or clinically significant adverse effects occur.

Therapy Background

Golodirsen (Vyondys 53) is an antisense oligonucleotide used for Duchenne muscular dystrophy (DMD) in patients with a confirmed DMD gene mutation amenable to exon 53 skipping. By promoting exon 53 skipping, the therapy enables production of dystrophin protein. The qualifying population is limited to members 15 years or younger at therapy initiation whose mutation is confirmed by genetic testing; coverage also requires the policy’s ambulatory, cardiac, pulmonary, corticosteroid, prescriber, and dosing criteria.

Policy Definitions

Mutation Amenable to Exon 53 Skipping

DefinitionA confirmed mutation of the DMD gene that can be treated by skipping exon 53

Treatment effectExon 53 skipping enables production of a dystrophin protein

DocumentationConfirmation by genetic testing is required for initial approval

6MWT

Abbreviation6MWT

Full term6-minute walk test

Policy useAssesses ambulatory function and required walking distance

Initial Authorization

Initial authorization requires that all criteria below are met.

Initial authorization

Disease and prescriber
- The member has Duchenne muscular dystrophy with a mutation amenable to exon 53 skipping, confirmed by genetic testing.
- Vyondys 53 is prescribed by or in consultation with a neurologist.
Age and clinical status
- The member is 15 years of age or younger at therapy initiation.Age ≤15 years

Continuation Authorization

Continuation authorization requires all criteria below, including either the standard objective-response pathway or the prior-insurer/stable-disease pathway.

Continuation authorization

The member is currently receiving medication for DMD with a mutation amenable to exon 53 skipping, or previously met the initial approval criteria.
One permitted positive-response pathway
- Standard objective-response pathway
  - Within the last 6 months, the member has ambulatory function, including the ability to walk with or without assistive devices, is not wheelchair dependent, and has a 6-minute walk test distance of at least 250 meters.6MWT ≥250 m

Quantity Limits

Quantity Limit

MedicationGolodirsen (Vyondys 53)

Maximum quantity30 mg/kg

FrequencyPer week; the dosing regimen is once weekly

Policy Summary

PayerWellcare Hawaii

PolicyGolodirsen (Vyondys 53) coverage for Duchenne muscular dystrophy

Policy CodePolicy CP.PHAR.453

Change TypeRevised

Effective Date03/01/2020

Last ReviewDec 1, 2020

Next Review DateN/A

Key ActionSubmit prior authorization documentation showing that all applicable initial or continuation approval criteria are met.

SourceLink

On This Page

Within the last 6 months, the member has stable pulmonary function with predicted forced vital capacity of at least 50%.Predicted FVC ≥50%

Prior-insurer/stable-disease pathway

The member received golodirsen through a healthcare insurer without meeting the standard response requirements.
The medical record shows improved or stable LVEF and FVC assessed within the last 6 months.Improved or stable LVEF and FVC within 6 months

The member has been assessed by a neurologist within the last 6 months.Neurologist assessment within 6 months

Vyondys 53 is prescribed concurrently with an oral corticosteroid, unless contraindicated or clinically significant adverse effects are experienced.

Vyondys 53 is not prescribed concurrently with another exon-skipping therapy, such as Exondys 51.

If the request is for a dose increase, the new dose does not exceed 30 mg/kg per week.≤30 mg/kg per week

Predicted Forced Vital Capacity

Required valueAt least 50% of predicted

Clinical statusStable pulmonary function

Assessment timingWithin the last 30 days for initial therapy and within the last 6 months for continuation therapy

Golodirsen Dose

Maximum dose30 mg/kg

FrequencyOnce weekly

AdministrationIntravenous (IV)

Vyondys 53 must be prescribed concurrently with an oral corticosteroid unless contraindicated or clinically significant adverse effects occur.

Documentation Required

Clinical Documentation Required

Submit office chart notes, laboratory results, and other clinical information supporting that the member meets all applicable approval criteria. Documentation should be current when the criteria specify a time window.

Include genetic testing, neurologic assessment, ambulatory function and 6-minute walk test results, cardiac function/LVEF, pulmonary function/FVC, corticosteroid history and response, medication regimen, and dose.
For continuation requests, include response measures assessed within the last 6 months and the neurologist assessment from within the last 6 months.

Documentation Required

Initial Therapy Records

For initial therapy, document Duchenne muscular dystrophy with a mutation amenable to exon 53 skipping, confirmed by genetic testing, and that the member is age 15 or younger when therapy begins. The prescription must be by or in consultation with a neurologist and must satisfy all clinical and dosing requirements.

Within the last 30 days, document ambulatory function with a 6MWT distance of at least 250 meters, LVEF greater than 50%, and predicted FVC of at least 50%.
Document inadequate response, shown by a significant decline in 6MWT, LVEF, or FVC, despite adherent oral corticosteroid use for at least 6 months, unless contraindicated or clinically significant adverse effects occur.
Document concurrent oral corticosteroid use unless contraindicated or clinically significant adverse effects occur; no concurrent exon-skipping therapy such as Exondys 51; and a dose not exceeding 30 mg/kg per week.

Documentation Required

Continuation Therapy Records

For continuation therapy, document current DMD treatment with a mutation amenable to exon 53 skipping or that the member previously met initial approval criteria. Demonstrate positive response and ongoing clinical oversight for the requested 6-month authorization period.

Within the last 6 months, document 6MWT distance of at least 250 meters, LVEF greater than 50%, and predicted FVC of at least 50%; alternatively, document improved or stable LVEF and FVC when the member received the medication through another insurer without meeting those measures.
Include a neurologist assessment from within the last 6 months.
Document concurrent oral corticosteroid use unless contraindicated or clinically significant adverse effects occur, no concurrent exon-skipping therapy such as Exondys 51, and any dose increase within 30 mg/kg per week.

Denial Risk

Initial Approval Criteria Must Be Met

An initial request may be denied when the required genetic confirmation, qualifying exon 53-skipping mutation, or other initial-therapy criteria are not documented or are not met.

Diagnosis of Duchenne muscular dystrophy and a mutation amenable to exon 53 skipping must be confirmed by genetic testing.
The member must be age 15 or younger at therapy initiation and the medication must be prescribed by or in consultation with a neurologist.
Required recent clinical measures must be met: 6MWT at least 250 meters, LVEF greater than 50%, and predicted FVC at least 50%.
The request must support inadequate response despite adherent oral corticosteroid use for at least 6 months, required concurrent oral corticosteroid use unless an exception applies, no concurrent exon-skipping therapy, and a dose no greater than 30 mg/kg per week.

Denial Risk

Continuation Criteria Must Be Met

Continuation authorization may be denied when the member does not demonstrate the required response, does not satisfy an applicable prior-insurer or stable-disease pathway, or lacks required ongoing documentation.

The member must show positive response through the required 6-month clinical measures or, under the alternative pathway, improved or stable LVEF and FVC documented within the last 6 months.
A neurologist assessment within the last 6 months is required.
Concurrent oral corticosteroid use, unless an exception applies, no concurrent exon-skipping therapy, and the dose limit for any increase must be documented.

Denial Risk

Unlisted or Off-Label Indication

Coverage is not authorized for diagnoses or indications that are not specifically listed in the policy. For an unlisted or off-label indication, refer to the applicable off-label use policy for the relevant line of business.

Commercial: CP.CPA.09
Health Insurance Marketplace: HIM.PHAR.21
Medicaid: CP.PMN.53

DMD

AbbreviationDMD

Full termDuchenne muscular dystrophy

Policy indicationTreatment is for DMD in patients with a confirmed mutation amenable to exon 53 skipping

FVC

AbbreviationFVC

Full termForced vital capacity

Policy usePredicted FVC must be at least 50%

LVEF

AbbreviationLVEF

Full termLeft ventricular ejection fraction

Policy useLVEF must be above 50% with stable cardiac function

Within the last 30 days, the member has ambulatory function, including the ability to walk with or without assistive devices, is not wheelchair dependent, and has a 6-minute walk test distance of at least 250 meters.6MWT ≥250 m

Within the last 30 days, the member has stable cardiac function with LVEF greater than 50%.LVEF >50%

Within the last 30 days, the member has stable pulmonary function with predicted FVC of at least 50%.Predicted FVC ≥50%

Corticosteroid criteria

The member has had an inadequate response, evidenced by a significant decline in 6MWT, LVEF, or FVC, despite adherent oral corticosteroid use for at least 6 months, unless contraindicated or clinically significantly harmful.Oral corticosteroid use ≥6 months
Vyondys 53 is prescribed concurrently with an oral corticosteroid, unless contraindicated or clinically significantly harmful.

Concurrent therapy and dosing

Vyondys 53 is not prescribed concurrently with another exon-skipping therapy, such as Exondys 51.
The dose does not exceed 30 mg/kg per week.≤30 mg/kg per week

Within the last 6 months, the member has stable cardiac function with LVEF greater than 50%.LVEF >50%

Within the last 6 months, the member has stable pulmonary function with predicted FVC of at least 50%.Predicted FVC ≥50%

Prior-insurer/stable-disease pathway

The member received golodirsen through a healthcare insurer without meeting the standard response requirements.
The medical record shows improved or stable LVEF and FVC assessed within the last 6 months.Improved or stable LVEF and FVC within 6 months

The member has been assessed by a neurologist within the last 6 months.Neurologist assessment within 6 months

Vyondys 53 is prescribed concurrently with an oral corticosteroid, unless contraindicated or clinically significantly harmful.

Vyondys 53 is not prescribed concurrently with another exon-skipping therapy, such as Exondys 51.

If the request is for a dose increase, the new dose does not exceed 30 mg/kg per week.≤30 mg/kg per week