CurrentAgency for Health Care AdministrationPolicy N/A

Brineura (cerliponase alfa) Prior Authorization Criteria

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This policy establishes prior authorization criteria for Brineura for patients with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2/TPP1 deficiency), including initial and continuation therapy requirements.

Policy Summary

PayerAgency for Health Care Administration

PolicyBrineura (cerliponase alfa) Prior Authorization Criteria

Policy CodePolicy N/A

Change TypeNo material clinical or coverage changes

Effective DateN/A

Next Review DateN/A

Key ActionSubmit prior authorization documentation confirming CLN2 diagnosis, baseline Motor domain assessment, and specialist prescribing.

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

1 yearAuthorization length

≥ 3 yearsMinimum patient age

4 levelsMotor rating levels

150 mg/5 mLSolution presentation

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CurrentAgency for Health Care AdministrationPolicy N/A

Brineura (cerliponase alfa) Prior Authorization Criteria

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Policy Summary

PayerAgency for Health Care Administration

PolicyBrineura (cerliponase alfa) Prior Authorization Criteria

Policy CodePolicy N/A

Change TypeNo material clinical or coverage changes

Effective DateN/A

Next Review DateN/A

Key ActionSubmit prior authorization documentation confirming CLN2 diagnosis, baseline Motor domain assessment, and specialist prescribing.

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

1 yearAuthorization length

≥ 3 yearsMinimum patient age

4 levelsMotor rating levels

150 mg/5 mLSolution presentation

Initial Therapy Criteria

Initial therapy is covered when ALL of the following are met.

Initial therapy

Eligibility and diagnosis
- Age: Patient is at least 3 years of age.≥ 3 years
- Confirmed CLN2 diagnosis
  - Enzyme testing: Official laboratory testing demonstrates deficient TPP1 enzyme activity in leukocytes, fibroblasts, or dried blood spots.
  - Genetic testing identifies causative mutations of the TPP1/CLN2 gene.
Baseline assessment: A baseline Motor domain CLN2 Clinical Rating Scale score is documented and the assessment tool is provided.
Specialist prescribing: The drug is prescribed by, or in consultation with, a neurologist or physician specializing in neuronal ceroid lipofuscinosis diseases.

Continuation Therapy Criteria

Continuation therapy is covered when ALL of the following are met.

Continuation therapy

Clinical response: Documentation shows improved clinical response with no signs of disease progression.
Motor score stability: The patient has not demonstrated a decline in the Motor domain of the CLN2 Clinical Rating Scale; current results or score must be documented.
Adverse-effect tolerance: The patient has not experienced treatment-restricting adverse effects.
Appropriate dosing: Dosing is appropriate according to product labeling or is supported by compendia.

Authorization and Documentation Requirements

Prior Authorization

Initial Authorization Requirements

Initial authorization requires documentation that the patient is at least 3 years of age and meets the clinical criteria for BRINEURA therapy. An authorization may be issued for 1 year, and the medication must be prescribed by or in consultation with a neurologist or physician specializing in neuronal ceroid lipofuscinosis diseases.

Patient must be ≥ 3 years of age.
Prescriber or consultant must be a neurologist or physician specializing in neuronal ceroid lipofuscinosis (NCL) diseases.
Length of authorization: 1 year.

Prior Authorization

Continuation Authorization

Continuation authorization requires documentation of an improved clinical response without signs of disease progression, together with continued eligibility for treatment. The patient must not have declined in the Motor domain of the CLN2 Clinical Rating Scale and must remain free of treatment-restricting adverse effects with appropriate dosing.

Improved clinical response with no signs of disease progression.
No decline in the Motor domain of the CLN2 Clinical Rating Scale.
No treatment-restricting adverse effects.
Dosing is consistent with labeling or supported by compendia.

Documentation Required

Diagnostic Confirmation Required

Submit documentation confirming a diagnosis of late infantile neuronal ceroid lipofuscinosis type 2 (CLN2), also known as tripeptidyl peptidase 1 (TPP1) deficiency. Confirmation must include deficient TPP1 enzyme activity demonstrated by an official laboratory in leukocytes, fibroblasts, or dried blood spots, or genetic testing identifying causative TPP1/CLN2 gene mutations.

Accepted enzyme-activity specimens: leukocytes, fibroblasts, or dried blood spots.
Alternatively, provide genetic testing results identifying causative mutations of the TPP1/CLN2 gene.

Documentation Required

Baseline Motor Assessment

Provide the baseline Motor domain score from a CLN2 Clinical Rating Scale before initial treatment. The submitted results must include the assessment tool used.

Score 3: Walks normally.
Score 2: Frequent falls; clumsiness evident.
Score 1: No unaided walking or only crawling.
Score 0: Immobile; mostly bedridden.

Documentation Required

Continuation Response Documentation

For continuation therapy, submit documentation of improved clinical response and no signs of disease progression, including current results for the Motor domain of the CLN2 Clinical Rating Scale. The current score must demonstrate that the patient has not declined from the documented baseline.

Provide current Motor domain assessment results and score.
Document improved clinical response and absence of disease progression.
Document no decline in the Motor domain score.

Denial Risk

Safety and Dosing Documentation

Continuation requests must document that treatment can proceed safely and is being administered appropriately. Authorization may be restricted when adverse effects prevent treatment or when dosing is not consistent with labeling or supported by compendia.

No treatment-restricting adverse effects.
Dosing is appropriate according to product labeling or supported by compendia.

Key Terms

CLN2

DefinitionLate infantile neuronal ceroid lipofuscinosis type 2

Also known asTripeptidyl peptidase 1 (TPP1) deficiency

Diagnostic confirmationConfirmed by official laboratory testing demonstrating deficient TPP1 enzyme activity in leukocytes, fibroblasts, or dried blood spots, or by genetic testing identifying causative TPP1/CLN2 gene mutations

CLN2 Clinical Rating Scale Motor domain

AssessmentFunctional motor assessment using the Motor domain of the CLN2 Clinical Rating Scale

Score rangeScores range from 3 to 0

Highest scoreScore 3 indicates the patient walks normally

Lowest scoreScore 0 indicates the patient is immobile and mostly bedridden

TPP1

Full nameTripeptidyl peptidase 1

Relationship to CLN2TPP1 is the enzyme deficient in CLN2 disease

Diagnostic relevanceDeficient TPP1 enzyme activity confirms CLN2, also known as TPP1 deficiency

Brineura Initial Therapy

Brineura Initial Criteria

Initial therapy requires ALL of the following.

Initial requirements

Age: Patient is at least 3 years of age.≥ 3 years
Confirmed CLN2/TPP1 deficiency
- Enzyme testing: Official laboratory testing demonstrates deficient TPP1 enzyme activity in leukocytes, fibroblasts, or dried blood spots.
- Genetic testing identifies causative mutations of the TPP1/CLN2 gene.
Baseline motor assessment: A baseline Motor domain CLN2 Clinical Rating Scale score is documented, and the assessment tool is provided.
Specialist prescribing: Treatment is prescribed by, or provided in consultation with, a neurologist or physician specializing in neuronal ceroid lipofuscinosis diseases.

Brineura (cerliponase alfa) Prior Authorization Criteria

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Brineura (cerliponase alfa) Prior Authorization Criteria

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Coverage Criteria

Clinical Rating Scale

Authorization and Documentation Requirements

Brineura and CLN2 Disease

Key Terms

Brineura Initial Therapy

Brineura Continuation Therapy