CurrentNew Hampshire Department of Health and Human ServicesPolicy N/A

Spinal Muscular Atrophy (SMA) Criteria

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This policy governs New Hampshire Medicaid Fee-for-Service prior authorization, quantity limits, approval durations, and renewal requirements for Evrysdi, Spinraza, Zolgensma, and Itvisma used to treat spinal muscular atrophy.

Policy Summary

PayerNew Hampshire Department of Health and Human Services

PolicySpinal Muscular Atrophy (SMA) Criteria

Policy CodePolicy N/A

Change TypeNo material change

Effective DateN/A

Next Review DateN/A

Key ActionSubmit prior approval requests with the drug-specific clinical criteria and supporting diagnostic, genetic, baseline assessment, laboratory, and administration documentation.

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

4SMA medications addressed

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CurrentNew Hampshire Department of Health and Human ServicesPolicy N/A

Spinal Muscular Atrophy (SMA) 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.

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Policy Summary

PayerNew Hampshire Department of Health and Human Services

PolicySpinal Muscular Atrophy (SMA) Criteria

Policy CodePolicy N/A

Change TypeNo material change

Effective DateN/A

Next Review DateN/A

Key ActionSubmit prior approval requests with the drug-specific clinical criteria and supporting diagnostic, genetic, baseline assessment, laboratory, and administration documentation.

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

4SMA medications addressed

Initial approval period for Evrysdi and Spinraza

1 yearRenewal approval period for Evrysdi and Spinraza

1 lifetimeApproval limit for Zolgensma and Itvisma

1 courseItvisma quantity limit

General Coverage Criteria

Evrysdi and Spinraza initial therapy

Evrysdi and Spinraza are covered when all of the following are met; the baseline assessment requirement may be satisfied by any one listed measure.

Evrysdi or Spinraza initial therapy

Diagnosis and treatment status
- Patient has a confirmed diagnosis of spinal muscular atrophy (SMA).
- Genetic testing demonstrates an SMN1 homozygous gene deletion or mutation.
- Patient is not concurrently receiving another treatment for SMA listed in the criteria.
ONE of the following baseline assessments
- Hammersmith Functional Motor Scale Expanded (HFMSE) is documented.
- Hammersmith Infant Neurologic Exam (HINE) is documented.
- Six-minute walk test (6MWT) is documented.
- Upper limb module (ULM) score is documented.
- Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) is documented.
- Bayley Scales of Infant and Toddler Development, Third Edition (BSID-III), is documented.
- Exacerbations requiring hospitalization and/or antibiotic therapy for respiratory infection in the previous year are documented.

Spinraza additional requirements

Spinraza has the Evrysdi and Spinraza initial therapy criteria plus all of the following additional requirements.

Spinraza-specific requirements

Quantitative spot urine protein testing is obtained at baseline and before each dose.
Complete blood count is obtained at baseline and before each dose.
Nusinersen is administered by a specialist with competency in intrathecal injection.

Zolgensma initial therapy

Zolgensma is covered when all of the following are met.

Patient is less than 2 years of age.less than 2 years
Patient has SMA confirmed by either bi-allelic deletion or dysfunctional point mutation of the SMN1 gene.
Patient has SMA confirmed by one to four copies of the SMN2 gene.one to four copies
Baseline anti-AAV9 antibody titer is at least 1:50, measured by ELISA.at least 1:50 by ELISA

Itvisma initial therapy

Itvisma is covered when all of the following are met.

Patient is at least 2 years of age.at least 2 years
Patient has SMA confirmed by either bi-allelic deletion or dysfunctional point mutation of the SMN1 gene.
Patient has SMA confirmed by one to four copies of the SMN2 gene.one to four copies
Baseline anti-AAV9 antibody titer is at least 1:50, measured by ELISA.at least 1:50 by ELISA

Evrysdi and Spinraza continuation therapy

Renewal of Evrysdi and Spinraza is covered when all of the following are met; the response requirement may be satisfied by any one listed measure.

There is no unacceptable toxicity from the drug.
Examples of unacceptable toxicity include serious infections, life-threatening glomerulonephritis, and thrombocytopenia.
ONE of the following response or stability measures
- Improvement or lack of progression is demonstrated by the Hammersmith Functional Motor Scale Expanded (HFMSE).
- Improvement or lack of progression is demonstrated by the Hammersmith Infant Neurologic Exam (HINE).
- Improvement or lack of progression is demonstrated by the Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND).

Zolgensma and Itvisma must not be used concurrently with nusinersen or risdiplam. Review the member’s current SMA therapy before requesting or administering either gene therapy.

Itvisma is not covered for a patient who has previously received Zolgensma. Confirm the member’s prior SMA gene-therapy history before requesting Itvisma.

Zolgensma coverage is limited to one dose and is not renewed after that single covered administration.

Clinical Coding and Thresholds

Baseline anti-AAV9 antibody titer

Measurement methodELISA

Minimum baseline titerAt least 1:50

Applies toZolgensma and Itvisma

Zolgensma patient age

MedicationZolgensma

Age requirementLess than 2 years of age

Coverage limitationOne dose; may not be renewed

Authorization and Documentation Requirements

Prior Authorization

Initial Prior Authorization Required

Prior approval is required before initiating treatment for spinal muscular atrophy. The request must identify the drug and include documentation supporting the applicable diagnosis, genetic testing, baseline assessments, and drug-specific clinical criteria.

Submit supporting clinical documentation with the prior authorization request.
Drug-specific criteria apply to Evrysdi, Spinraza, Zolgensma, and Itvisma.

Prior Authorization

Renewal Prior Authorization

Renewal prior authorization applies to Evrysdi and Spinraza only. The renewal request must document continued eligibility, absence of unacceptable toxicity, and improvement or lack of progression on at least one accepted clinical or functional measure.

Document the patient's response or non-progression since the prior approval.
Report unacceptable toxicity, including serious infections, life-threatening glomerulonephritis, or thrombocytopenia.

Spinal Muscular Atrophy Overview

Spinal muscular atrophy (SMA) is a genetic neuromuscular disorder associated with abnormalities of the SMN1 gene and progressive loss of motor neurons. The policy’s coverage requirements therefore focus on confirmation of the genetic diagnosis, baseline functional or respiratory status, treatment safety monitoring, and appropriate selection and administration of SMA therapies.

Policy Definitions

Baseline assessment

Required documentationAt least one qualifying baseline measure

Motor or developmental measuresHFMSE, HINE, 6MWT, ULM score, CHOP INTEND, or BSID-III

Respiratory-infection measureExacerbations requiring hospitalization and/or antibiotic therapy for respiratory infection in the previous year

Anti-AAV9 antibody titer

DefinitionLaboratory measurement of antibodies against adeno-associated virus serotype 9

Testing methodELISA

Baseline threshold

Initial Therapy by Medication

Evrysdi and Spinraza — initial therapy criteria

Evrysdi and Spinraza initial therapy is covered when all of the following are met.

Evrysdi and Spinraza initial therapy

Diagnosis and genetic confirmation
- Patient has documentation of a confirmed diagnosis of spinal muscular atrophy (SMA).
- Genetic testing demonstrates an SMN1 homozygous gene deletion or mutation.
Treatment status
- Patient is not concurrently receiving another treatment for SMA listed in the criteria.
ONE qualifying baseline assessment

Renewal Therapy

Evrysdi and Spinraza renewal

Renewal of Evrysdi and Spinraza is covered when all of the following are met.

There is an absence of unacceptable toxicity from the drug.
Examples of unacceptable toxicity include serious infections, life-threatening glomerulonephritis, and thrombocytopenia.
ONE qualifying improvement or stability measure
- Improvement or lack of progression is demonstrated by Hammersmith Functional Motor Scale Expanded (HFMSE).
- Improvement or lack of progression is demonstrated by Hammersmith Infant Neurologic Exam (HINE).
- Improvement or lack of progression is demonstrated by Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND).

Medication Quantity Limits

Evrysdi oral solution quantity limit

FormulationOral solution

Quantity limit180 mg (240 mL; 3 bottles)

PeriodPer 30 days

Evrysdi tablets quantity limit

Formulation5 mg tablets

Quantity limitOne tablet daily

PeriodPer day

Administration Site and Specialist Requirements

Billing Rule

Use a qualified nusinersen specialist

Nusinersen must be administered by a specialist with competency in intrathecal injection.

Billing Rule

Use a qualified Itvisma specialist

Itvisma must be administered by a specialist with competency in intrathecal injection.

Policy Summary

PayerNew Hampshire Department of Health and Human Services

PolicySpinal Muscular Atrophy (SMA) Criteria

Policy CodePolicy N/A

Change TypeNo material change

Effective DateN/A

Next Review DateN/A

Key ActionSubmit prior approval requests with the drug-specific clinical criteria and supporting diagnostic, genetic, baseline assessment, laboratory, and administration documentation.

SourceLink

On This Page

Patient does not have pre-existing hepatic impairment as assessed by pretreatment liver function tests, including total bilirubin, prothrombin time, AST, and ALT.

Patient does not have advanced disease, such as complete limb paralysis or permanent ventilation support.

Onasemnogene abeparvovec-xioi is used concomitantly with parenteral corticosteroids.

Onasemnogene abeparvovec-xioi is not used in combination with nusinersen or risdiplam.

Coverage is provided for one dose and may not be renewed.

One administration per lifetime.

Patient does not have pre-existing hepatic impairment as assessed by pretreatment liver function tests, including total bilirubin, prothrombin time, AST, and ALT.

Patient baseline platelet counts are assessed, with monitoring weekly for the first month and as indicated until the post-treatment platelet count returns to baseline.

Patient does not have advanced disease, such as complete limb paralysis or permanent ventilation support.

Onasemnogene abeparvovec-brve is used concomitantly with systemic corticosteroids.

Onasemnogene abeparvovec-brve is not used in combination with nusinersen or risdiplam.

Patient has not received a treatment with SMA gene therapy, including Zolgensma.

Treatment is administered by a specialist with competency in intrathecal injection.

Improvement or lack of progression is demonstrated by the Bayley Scales of Infant and Toddler Development, Third Edition (BSID-III).

Improvement or lack of progression is demonstrated by the six-minute walk test (6MWT).

Improvement or lack of progression is demonstrated by the upper limb module (ULM) score.

Improvement or lack of progression is demonstrated by respiratory function tests.

Improvement or lack of progression is demonstrated by patient weight.

Exacerbations requiring hospitalization and/or antibiotic therapy for respiratory infection in the previous year are documented without progression.

Itvisma patient age

MedicationItvisma

Age requirementAt least 2 years of age

Administration requirementAdministered by a specialist with competency in intrathecal injection

SMN2 gene copy requirement

GeneSMN2

Required copy numberOne to four copies

Applies toZolgensma and Itvisma

Renewal duration is subject to the approved authorization period and continued satisfaction of the renewal criteria.

Documentation Required

Evrysdi and Spinraza Diagnostic and Baseline Documentation

For Evrysdi or Spinraza, document a confirmed diagnosis of SMA and genetic testing demonstrating an SMN1 homozygous gene deletion or mutation. The request must also include a baseline assessment using at least one accepted motor, developmental, respiratory, or infection-related measure.

Accepted baseline measures include HFMSE, HINE, 6MWT, ULM, CHOP INTEND, BSID-III, or exacerbations requiring hospitalization and/or antibiotic therapy for respiratory infection during the previous year.

Documentation Required

Spinraza Monitoring and Administration

Spinraza requests must include the monitoring and administration requirements specific to nusinersen. Quantitative spot urine protein testing and a complete blood count are required at baseline and before each dose.

Nusinersen must be administered by a specialist with competency in intrathecal injection.
Document quantitative spot urine protein results before each dose.
Document complete blood count results before each dose.

Documentation Required

Zolgensma Clinical Documentation

For Zolgensma, document SMA confirmed by bi-allelic SMN1 deletion or dysfunctional point mutation and one to four copies of SMN2. The patient must have a baseline anti-AAV9 antibody titer of at least 1:50 by ELISA, no pre-existing hepatic impairment based on pretreatment liver function tests, and no advanced disease.

Pretreatment liver function documentation must include total bilirubin, prothrombin time, AST, and ALT.
Advanced disease includes examples such as complete limb paralysis or permanent ventilation support.
Document concomitant use of parenteral corticosteroids.

Documentation Required

Itvisma Clinical Documentation

For Itvisma, document SMA confirmed by bi-allelic SMN1 deletion or dysfunctional point mutation and one to four copies of SMN2. The request must include a baseline anti-AAV9 antibody titer of at least 1:50 by ELISA, pretreatment hepatic assessment, platelet monitoring, and evidence that the patient does not have advanced disease.

Pretreatment liver function documentation must include total bilirubin, prothrombin time, AST, and ALT.
Assess baseline platelet counts and monitor weekly during the first month and as indicated until the post-treatment platelet count returns to baseline.
Document concomitant use of systemic corticosteroids.
Itvisma must be administered by a specialist with competency in intrathecal injection.

Documentation Required

Renewal Response Documentation

Renewal requests for Evrysdi and Spinraza must document absence of unacceptable toxicity and improvement or lack of progression in at least one accepted measure.

Accepted response measures include HFMSE, HINE, CHOP INTEND, BSID-III, 6MWT, ULM score, respiratory function tests, patient weight, or respiratory infection exacerbations requiring hospitalization and/or antibiotic therapy during the previous year.

Denial Risk

Failure to Meet Approval Criteria

Prior approval will be denied when the applicable approval criteria are not met.

Ensure every required clinical, genetic, laboratory, monitoring, and administration criterion is documented before submission.

Denial Risk

Concurrent SMA Therapy Prohibited

Concurrent treatment with another SMA therapy listed in the criteria is not permitted. Zolgensma and Itvisma must not be used in combination with nusinersen or risdiplam, and Evrysdi or Spinraza patients may not concurrently receive another listed SMA treatment.

Do not combine Zolgensma or Itvisma with Spinraza or Evrysdi.
Document that the patient is not receiving another SMA treatment listed in the criteria.

Denial Risk

Prior Gene Therapy or Repeat Administration

Gene therapy coverage is limited to a single administration and is not repeatable. Zolgensma coverage is provided for one dose and may not be renewed; Itvisma is not covered for a patient who previously received Zolgensma.

Document prior SMA gene-therapy history before requesting Itvisma.
Do not submit a renewal request for a Zolgensma dose.

Documentation Required

Product-Specific Age Requirements

Age thresholds differ by product. Zolgensma requires the patient to be less than 2 years of age, while Itvisma requires the patient to be at least 2 years of age.

Zolgensma: less than 2 years of age.
Itvisma: at least 2 years of age.

At least 1:50 for Zolgensma and Itvisma

Hammersmith Functional Motor Scale Expanded (HFMSE) is documented.
Hammersmith Infant Neurologic Exam (HINE) is documented.
Six-minute walk test (6MWT) is documented.
Upper limb module (ULM) score is documented.
Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) is documented.
Bayley Scales of Infant and Toddler Development, Third Edition (BSID-III), is documented.
Exacerbations requiring hospitalization and/or antibiotic therapy for respiratory infection in the previous year are documented.

Zolgensma — initial therapy criteria

Zolgensma initial therapy is covered when all of the following are met.

Zolgensma criteria

Age and genetic criteria
- Patient is less than 2 years of age.less than 2 years
- SMA is confirmed by either bi-allelic deletion or dysfunctional point mutation of the SMN1 gene.
- SMA is confirmed by one to four copies of the SMN2 gene.one to four copies
Laboratory and disease-status criteria
- Baseline anti-AAV9 antibody titer is at least 1:50, measured by ELISA.at least 1:50 by ELISA
- Patient does not have pre-existing hepatic impairment based on pretreatment liver function tests, including total bilirubin, prothrombin time, AST, and ALT.
- Patient does not have advanced disease, such as complete limb paralysis or permanent ventilation support.
Corticosteroid and combination-therapy criteria
- Onasemnogene abeparvovec-xioi is used concomitantly with parenteral corticosteroids.
- Onasemnogene abeparvovec-xioi is not used in combination with nusinersen or risdiplam.
Coverage is provided for one dose and may not be renewed.
One administration per lifetime.

Itvisma — initial therapy criteria

Itvisma initial therapy is covered when all of the following are met.

Itvisma criteria

Age and genetic criteria
- Patient is at least 2 years of age.at least 2 years
- SMA is confirmed by either bi-allelic deletion or dysfunctional point mutation of the SMN1 gene.
- SMA is confirmed by one to four copies of the SMN2 gene.one to four copies
Laboratory and disease-status criteria
- Baseline anti-AAV9 antibody titer is at least 1:50, measured by ELISA.at least 1:50 by ELISA
- Patient does not have pre-existing hepatic impairment based on pretreatment liver function tests, including total bilirubin, prothrombin time, AST, and ALT.
- Patient baseline platelet counts are assessed, with monitoring weekly for the first month and as indicated until the post-treatment platelet count returns to baseline.
- Patient does not have advanced disease, such as complete limb paralysis or permanent ventilation support.
Treatment and administration criteria
- Onasemnogene abeparvovec-brve is used concomitantly with systemic corticosteroids.
- Onasemnogene abeparvovec-brve is not used in combination with nusinersen or risdiplam.
- Patient has not received a treatment with SMA gene therapy, including Zolgensma.
- Treatment is administered by a specialist with competency in intrathecal injection.

Improvement or lack of progression is demonstrated by Bayley Scales of Infant and Toddler Development, Third Edition (BSID-III).

Improvement or lack of progression is demonstrated by six-minute walk test (6MWT).

Improvement or lack of progression is demonstrated by upper limb module (ULM) score.

Improvement or lack of progression is demonstrated by respiratory function tests.

Improvement or lack of progression is demonstrated by patient weight.

Exacerbations requiring hospitalization and/or antibiotic therapy for respiratory infection in the previous year are documented.

Spinraza initial therapy quantity limit

Therapy phaseInitial therapy

Quantity limitFour vials

Coverage periodFor the first 58 days

Spinraza maintenance quantity limit

Therapy phaseMaintenance

Quantity limitOne vial

IntervalEvery 120 days

Zolgensma quantity limit

MedicationZolgensma

Quantity limit1 kit

Quantity basisPer course

Itvisma quantity limit

MedicationItvisma

Quantity limit1 treatment course

Quantity basisPer course