CurrentNew Hampshire Department of Health and Human ServicesPolicy N/A

Skysona (elivaldogene autotemcel) Coverage Criteria

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This policy governs prior authorization and coverage of Skysona (elivaldogene autotemcel) for New Hampshire Medicaid fee-for-service beneficiaries. It applies to boys aged 4 through 17 years with early, active cerebral adrenoleukodystrophy who meet specified clinical, laboratory, infection-screening, and treatment-planning requirements.

Policy Summary

PayerNew Hampshire Department of Health and Human Services

PolicySkysona (elivaldogene autotemcel) Coverage Criteria

Policy CodePolicy N/A

Change TypeNo material change

Effective DateN/A

Next Review DateN/A

Key ActionSubmit prior authorization documentation confirming that all clinical, laboratory, safety, and exclusion criteria are satisfied.

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

4 to <18 yearsEligible patient age range

Loes 0.5-9Required MRI disease activity range

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CurrentNew Hampshire Department of Health and Human ServicesPolicy N/A

Skysona (elivaldogene autotemcel) Coverage Criteria

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

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Policy Summary

PayerNew Hampshire Department of Health and Human Services

PolicySkysona (elivaldogene autotemcel) Coverage Criteria

Policy CodePolicy N/A

Change TypeNo material change

Effective DateN/A

Next Review DateN/A

Key ActionSubmit prior authorization documentation confirming that all clinical, laboratory, safety, and exclusion criteria are satisfied.

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

4 to <18 yearsEligible patient age range

Loes 0.5-9Required MRI disease activity range

Maximum neurological function score

1 doseLifetime treatment limitation

≥5 × 10^6 CD34+ cells/kgMinimum dose

Coverage Criteria

Initial Therapy Criteria

Covered when all of the following criteria are met.

All approval criteria

Patient and diagnosis
- CALD confirmation: The patient has elevated very-long-chain fatty acid (VLCFA) values and a confirmed ABCD1 mutation detected by molecular genetic testing.C26:0: 1.30 ± 0.45; C24:0/C22:0: 1.71 ± 0.23; C26:0/C22:0: 0.07 ± 0.03
Active central nervous system disease
- MRI findings: The MRI demonstrates a Loes score between 0.5 and 9, inclusive, on the 34-point scale, and gadolinium enhancement on demyelinating lesions.Loes score 0.5-9 inclusive; gadolinium enhancement present
Neurologic status: The patient has a neurological function score (NFS) less than or equal to 1.NFS ≤1
Represents asymptomatic or mildly symptomatic disease.
Infectious-disease screening and safety: The patient has been screened for hepatitis B virus, hepatitis C virus, human T-lymphotrophic virus 1 and 2, and human immunodeficiency virus 1 and 2 in accordance with clinical guidelines before collection of cells by leukapheresis; does not have an active infection, including a clinically important localized infection; and will follow infection prophylaxis according to standard institutional guidelines.HBV, HCV, HTLV-1/HTLV-2, and HIV-1/HIV-2 screening before leukapheresis
Vaccination requirements: Vaccinations will not be administered within the six weeks before the start of therapy or concurrently while on therapy, and the patient is up to date with all age-appropriate vaccinations in accordance with current vaccination guidelines before initiating therapy.No vaccination within 6 weeks before therapy or concurrently during therapy
Long-term monitoring: The patient will receive periodic lifelong monitoring for hematological malignancies.
Antiretroviral medication restriction: The patient will avoid concomitant therapy with antiretroviral medications for at least one month before initiating medications for stem-cell mobilization, for the expected duration of medication elimination, and until all cycles of apheresis are completed.At least 1 month before stem-cell mobilization through completion of apheresis and the expected elimination period
Exclusions absent: The disease is not head-trauma-induced; therapy will not be used to prevent the development of or treat adrenal insufficiency due to adrenoleukodystrophy; the patient has no history of hematopoietic stem cell transplant; and the patient has no known or available HLA-matched willing family donor.

Head-trauma-induced disease is excluded from coverage under this policy.

Skysona is not covered when used to prevent the development of or treat adrenal insufficiency due to adrenoleukodystrophy.

A history of hematopoietic stem cell transplant excludes the patient from coverage.

Coverage is excluded when the patient has a known or available human leukocyte antigen (HLA)-matched willing family donor.

The patient must not have an active infection, including a clinically important localized infection, for coverage to be approved.

Clinical Coding and Thresholds

Very-long-chain fatty acids (VLCFA)

C26:01.30 ± 0.45 (normal: 0.23 ± 0.09)

C24:0/C22:01.71 ± 0.23 (normal: 0.84 ± 0.10)

C26:0/C22:00.07 ± 0.03 (normal: 0.01 ± 0.004)

MRI Loes score

Assessment methodBrain MRI using the 34-point Loes scale

Qualifying rangeLoes score from 0.5 through 9, inclusive

Disease findingActive central nervous system disease established by central radiographic review

Provider Actions and Authorization Requirements

Prior Authorization

Prior Authorization Required

Prior authorization is required before Skysona (elivaldogene autotemcel) is provided. Submit documentation demonstrating that all approval criteria are satisfied.

Documentation Required

Disease and Neurologic Documentation

Document the patient’s disease and neurologic eligibility for treatment, including age, early active CALD diagnosis, elevated VLCFA values, confirmed ABCD1 mutation, active MRI-confirmed CNS disease, and neurologic status.

Male patient is at least 4 years old and younger than 18 years old.
Documented early, active cerebral adrenoleukodystrophy (CALD).
Elevated VLCFA values: C26:0 1.30 ± 0.45; C24:0/C22:0 1.71 ± 0.23; and C26:0/C22:0 0.07 ± 0.03.
Confirmed ABCD1 mutation detected by molecular genetic testing.

Clinical Background

Skysona (elivaldogene autotemcel) is used to slow the progression of neurologic dysfunction in boys aged 4 through 17 years with early, active cerebral adrenoleukodystrophy (CALD). Eligibility requires biochemical and molecular confirmation of CALD, active central nervous system demyelinating disease on MRI, and asymptomatic or mildly symptomatic neurologic disease. Treatment also requires infectious-disease screening before leukapheresis, infection and vaccination precautions, and lifelong monitoring for hematologic malignancies.

Key Definitions

Cerebral adrenoleukodystrophy (CALD)

PopulationBoys 4 through 17 years of age

DiseaseEarly, active cerebral adrenoleukodystrophy (CALD), with active CNS disease established by brain MRI

Treatment purposeTo slow the progression of neurologic dysfunction

Loes score

ScaleA 34-point MRI-based scale

Qualifying rangeLoes score from 0.5 through 9, inclusive

Additional MRI findingGadolinium enhancement on MRI of demyelinating lesions is required

Initial Therapy

Initial Therapy Criteria

Initial therapy is covered only when all listed clinical, laboratory, safety, treatment-planning, and exclusion criteria are satisfied.

Initial Skysona eligibility

Clinical and laboratory eligibility: The patient is male, is 4 years of age or older and younger than 18 years of age, has documented early, active CALD, has elevated VLCFA values, and has a confirmed ABCD1 mutation detected by molecular genetic testing.Age 4 to <18 years; C26:0: 1.30 ± 0.45; C24:0/C22:0: 1.71 ± 0.23; C26:0/C22:0: 0.07 ± 0.03
Active CNS disease: Central radiographic review of brain MRI demonstrates active CNS disease with a Loes score between 0.5 and 9, inclusive, on the 34-point scale, and gadolinium enhancement on demyelinating lesions.Loes score 0.5-9 inclusive; gadolinium enhancement present

Quantity and Lifetime Limits

Skysona (elivaldogene autotemcel)

DoseA single dose

Cell quantityMinimum of 5 × 10^6 CD34+ cells/kg of body weight

Infusion packagingAdministered in one or more infusion bags

Lifetime limitLimited to one dose per lifetime

Policy Summary

PayerNew Hampshire Department of Health and Human Services

PolicySkysona (elivaldogene autotemcel) Coverage Criteria

Policy CodePolicy N/A

Change TypeNo material change

Effective DateN/A

Next Review DateN/A

Key ActionSubmit prior authorization documentation confirming that all clinical, laboratory, safety, and exclusion criteria are satisfied.

SourceLink

On This Page

Neurological function score

Maximum qualifying scoreNFS ≤1

Score 0Qualifying neurological function score for this policy

Score 1Qualifying neurological function score for this policy

Central radiographic review of brain MRI demonstrates a Loes score of 0.5–9, inclusive, on the 34-point scale, with gadolinium enhancement of demyelinating lesions.

Neurological function score (NFS) is less than or equal to 1, indicating asymptomatic or mildly symptomatic disease.

Documentation Required

Safety and Treatment-Readiness Documentation

Document that required infection screening and treatment-readiness measures are complete before cell collection and therapy initiation.

Screening for HBV, HCV, HTLV-1/HTLV-2, and HIV-1/HIV-2 has been completed in accordance with clinical guidelines before leukapheresis.
Patient does not have an active infection, including a clinically important localized infection.
Infection prophylaxis will be followed according to standard institutional guidelines.
Patient is up to date with age-appropriate vaccinations before therapy; vaccinations will not be administered during the 6 weeks before therapy or concurrently with therapy.

Documentation Required

Treatment-Planning Documentation

Document the treatment plan and confirm that required therapy-avoidance, monitoring, and donor-related requirements are satisfied before authorization.

Patient will avoid concomitant antiretroviral therapy for at least 1 month before medications for stem-cell mobilization, throughout the expected medication-elimination period, and until all apheresis cycles are completed.
Patient will receive periodic lifelong monitoring for hematologic malignancies.
Patient has no history of hematopoietic stem-cell transplant.
There is no known or available HLA-matched willing family donor.

Denial Risk

Failure to Meet Approval Criteria

Coverage is denied when the approval criteria are not met. The provider must submit sufficient clinical documentation to establish that every required criterion is satisfied.

All criteria for age, diagnosis, laboratory findings, genetic confirmation, MRI disease activity, neurologic status, infection screening, treatment readiness, and treatment planning must be met.

Denial Risk

Excluded Clinical Circumstances

Skysona is not approved when an excluded clinical circumstance applies.

Disease is induced by head trauma.
Therapy is requested to prevent or treat adrenal insufficiency due to adrenoleukodystrophy.
Patient has a history of hematopoietic stem-cell transplant.
A known or available HLA-matched willing family donor exists.

Denial Risk

Clinical Eligibility Requirements Not Met

Coverage is not approved when the patient has an active infection or otherwise fails to satisfy the clinical eligibility requirements for Skysona.

Active infection, including a clinically important localized infection, is present.
Any required clinical eligibility criterion is not satisfied or cannot be documented.

Neurological function score (NFS)

Qualifying score 0Represents asymptomatic or mildly symptomatic disease for purposes of this policy

Qualifying score 1Represents asymptomatic or mildly symptomatic disease for purposes of this policy

Policy thresholdNFS less than or equal to 1

Neurologic function: The patient has a neurological function score less than or equal to 1.NFS ≤1

Asymptomatic or mildly symptomatic disease.

Infection screening and precautions: Before leukapheresis, the patient has been screened for HBV, HCV, HTLV-1/HTLV-2, and HIV-1/HIV-2 in accordance with clinical guidelines; the patient has no active infection, including a clinically important localized infection; and infection prophylaxis will follow standard institutional guidelines.

Vaccination and monitoring plan: Vaccinations will not be administered within six weeks before therapy or concurrently during therapy; age-appropriate vaccinations are current before therapy; and the patient will receive periodic lifelong monitoring for hematological malignancies.

Treatment planning: The patient will avoid concomitant antiretroviral therapy for at least one month before initiating stem-cell mobilization medications, for the expected duration of medication elimination, and until all apheresis cycles are completed.

Required exclusions absent: The disease is not head-trauma-induced; treatment is not intended to prevent or treat adrenal insufficiency due to adrenoleukodystrophy; the patient has no history of hematopoietic stem cell transplant; and there is no known or available HLA-matched willing family donor.