CurrentCareSourcePolicy N/A

UTILIZATION MANAGEMENT MEDICAL POLICY

Defines prior authorization recommendation and medical necessity criteria, dosing, prescribing specialist requirements, and exclusions for Brineura (cerliponase alfa intraventricular infusion) for treatment of neuronal ceroid lipofuscinosis type 2 (CLN2). Applies to medical-benefit coverage decisions.

Policy Summary

PayerCareSource

PolicyUTILIZATION MANAGEMENT MEDICAL POLICY

Policy CodePolicy N/A

Change TypeRevised criteria and dosing

Effective Date

Next Review Date

Key ActionPrior Authorization is recommended; approval requires meeting the listed diagnostic, specialist involvement, and dosing criteria.

SourceLink

POLICY UPDATE CHANGES

Requirement changed to require two pathogenic variants in CLN2 gene plus reduced TPP1 activity for diagnosis confirmation (previous wording differed).

Dosing schedule updated to include age-based dosing from birth through adulthood up to 22 years and specific loading/maintenance for 1 to <2 years.

Removed requirement that each Brineura dose be followed by an infusion of intraventricular electrolytes.

1Covered Indication (CLN2 disease)

4Age-based dosing tiers

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Coverage Summary

Defines prior authorization recommendation and medical necessity criteria for Brineura (cerliponase alfa) to treat CLN2 disease. Coverage is covered_with_criteria when diagnostic confirmation (two pathogenic variants in CLN2 plus reduced TPP1 activity) and specialist prescribing/consultation requirements are met. Background notes that Brineura is given via intracerebroventricular (ICV) infusion through a surgically implanted reservoir and catheter and carries a Boxed Warning for hypersensitivity including anaphylaxis.

Quick facts

IndicationBrineura (cerliponase alfa) for CLN2 disease to slow loss of ambulation in symptomatic pediatric patients

Coverage stancecovered_with_criteria

SettingMedical benefit (ICV infusion via surgically implanted reservoir and catheter)

Population notesAge-based dosing tiers (4 tiers): Birth to <6 months; 6 months to <1 year; 1 year to <2 years (loading 200 mg ×4 then 300 mg QOW); 2 years to 22 years (300 mg QOW)

Diagnostic requirementsGenetic testing confirming two pathogenic variants in CLN2 PLUS a test confirming reduced TPP1 enzyme activity (e.g., leukocytes, fibroblasts, or dried blood spot)

Medical-Necessity Criteria

Provider Actions

Prior Authorization

Prior authorization required

Prior Authorization is recommended for medical benefit coverage of Brineura; approval requires meeting the listed criteria and dosing and requires prescribing by or in consultation with an appropriate specialist.

Documentation Required

Diagnostic confirmation required

Documentatory evidence must include genetic testing confirming two pathogenic variants in CLN2 and a test confirming reduced TPP1 enzyme activity.

Genetic testing confirming two pathogenic variants in the CLN2 gene
Reduced TPP1 enzyme activity in leukocytes, fibroblasts, or dried blood spot

Documentation Required

Coding

Drug (NDC) - not listed in documentNDC

No codes listed

Administration (CPT/HCPCS) - ICV infusion - not specified in documentCPT|HCPCS

No codes listed

Background

Brineura (cerliponase alfa) is a recombinant human tripeptidyl peptidase 1 (TPP1) indicated to slow the loss of ambulation in symptomatic pediatric patients with neuronal ceroid lipofuscinosis type 2 (CLN2). It is administered via intracerebroventricular (ICV) infusion into the cerebrospinal fluid through a surgically implanted reservoir and catheter and should be given by or under the direction of a physician experienced in ICV administration.

Safety includes a Boxed Warning for hypersensitivity reactions including anaphylaxis; initiation should occur in a health care setting with appropriate monitoring. Mechanistically, Brineura is recombinant human TPP1 produced by recombinant DNA technology and replaces deficient enzyme activity specific to CLN2 disease.

Evidence: Prescribing information — Brineura intraventricular infusion prescribing information, BioMarin; July 2024.

Definitions

TPP1Tripeptidyl peptidase 1 enzyme; deficiency causes CLN2 disease

CLN2Neuronal ceroid lipofuscinosis type 2 (also known as late infantile NCL or TPP1 deficiency)

Revision History

08/07/2024revised

Requirement changed to require two pathogenic variants in CLN2 gene plus reduced TPP1 activity for diagnosis confirmation (previous wording differed).

08/07/2024revised

Dosing schedule updated to include age-based dosing from birth through adulthood up to 22 years and specific loading/maintenance for 1 to <2 years.

08/07/2024revised

Removed requirement that each Brineura dose be followed by an infusion of intraventricular electrolytes.