CurrentmhnPolicy CP.PHAR.338

Cerliponase Alfa (Brineura)

Defines medical necessity, approval criteria, dosing, and exclusions for Brineura (cerliponase alfa) for treatment of neuronal ceroid lipofuscinosis type 2 (CLN2) for health plans affiliated with Centene Corporation; applies to commercial, HIM and Medicaid lines of business.

Policy Summary

Payermhn

PolicyCerliponase Alfa (Brineura)

Policy CodePolicy CP.PHAR.338

Change TypeRevised criteria and added coding

Effective DateJul 1, 2017

Next Review Date

Key ActionObtain prior authorization and submit documentation confirming diagnosis, neurologist involvement, weight ≥ 2.5 kg, enzyme and genetic testing, and motor score.

SourceLink

POLICY UPDATE CHANGES

Updated criteria to reflect FDA-approved indication expansion to include symptomatic and presymptomatic patients younger than 3 years and replaced prior age requirement with a minimum weight requirement of 2.5 kg; added boxed warning for hypersensitivity including anaphylaxis.

Added HCPCS code J0567 for injection, cerliponase alfa, 1 mg.

300 mgmaximum dose every other week

6 moapproval duration

≥ 2.5 kgminimum member weight

J0567HCPCS code

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Coverage Criteria

Initial Therapy — Covered when ALL of the following are met

Covered when ALL of the following are met:

Initial therapy for CLN2: Member has a diagnosis of neuronal ceroid lipofuscinosis type 2 (CLN2).

Prescribing clinician: Medication is prescribed by or in consultation with a neurologist.

Minimum weight: Member weighs >= 2.5 kg.

Confirmatory testing: Confirmation of CLN2 with BOTH of the following: (a) TPP1 enzyme activity test demonstrating deficient TPP1 enzyme activity in leukocytes; and (b) identification of two pathogenic mutations in trans in the TPP1/CLN2 gene.

Baseline function: Motor domain of the CLN2 Clinical Rating Scale score >= 1 (see Appendix D).

Device / institutional status: At the time of request, member does not have acute intraventricular access device–related complications (e.g., leakage, device failure, or device-related infection) or ventriculoperitoneal shunts.

Dosing plan: Planned dose does not exceed 300 mg administered once every other week as an intraventricular infusion.

Continuation Therapy — Continued coverage when ALL of the following are met

Continued coverage when ALL of the following are met:

Eligibility at time of renewal: Member is currently receiving medication via the Centene benefit and/or previously met initial approval criteria, or is in a state/product with continuity of care regulations allowing continued therapy.

Clinical benefit or stabilization: Member is responding positively to therapy or demonstrating stabilization as evidenced by a score >= 1 on the CLN2 Clinical Rating Scale motor domain.

Device / institutional status: Member does not have acute intraventricular access device–related complications (e.g., leakage, device failure, or device-related infection) or ventriculoperitoneal shunts.

Dosing adherence: If the request is for a dose increase, the new dose does not exceed 300 mg administered once every other week as an intraventricular infusion.

Other Indications — Requests for other diagnoses or indications

Requests for other diagnoses or indications:

Other indications: If the drug has recently (within the last 6 months) undergone a label change not yet reflected in this policy, follow the specified formulary or non‑formulary policy pathways for the relevant line of business (see policy references).

Off‑label requests: If the requested use is NOT specifically listed under 'Diagnoses/Indications for which coverage is NOT authorized' and the label‑change pathway does not apply, refer the request to the applicable off‑label use policy for the relevant line of business (CP.CPA.09, HIM.PA.154, or CP.PMN.53).

Not medically necessary / denial logic: Uses that are non‑FDA approved and lack sufficient documentation of efficacy and safety per the applicable off‑label use policy are considered not authorized. Requests may be denied when required documentation (e.g., diagnosis confirmation, neurologist involvement, confirmatory enzyme and genetic testing, minimum weight, motor score, and appropriate device status) is not provided. Refer to policy exclusions for contraindications (e.g., acute unresolved device‑site infection, suspected/confirmed CNS infection, acute intraventricular access device complications, or ventriculoperitoneal shunts).

This policy covers Brineura (cerliponase alfa) only for FDA‑approved indications addressed herein. Non‑FDA approved indications that are not specifically addressed in this policy are not authorized unless sufficient documentation of efficacy and safety is provided in accordance with the applicable off‑label use policy: CP.CPA.09 (Commercial), HIM.PA.154 (Health Insurance Marketplace), or CP.PMN.53 (Medicaid), or unless coverage is supported by the plan’s evidence of coverage documents.

Contraindications to intraventricular administration of cerliponase alfa include: acute, unresolved localized infection on or around the device insertion site (for example, cellulitis or abscess), suspected or confirmed central nervous system infection (e.g., cloudy CSF, positive CSF gram stain, or meningitis), acute intraventricular access device‑related complications (such as leakage, device failure, or device‑related infection), and patients with ventriculoperitoneal shunts. The product also carries a boxed warning for hypersensitivity reactions including anaphylaxis.

Requests for use of cerliponase alfa for indications that are not FDA‑approved and are not described in this policy are not authorized unless the provider submits sufficient clinical documentation to meet the applicable off‑label use policy requirements (see CP.CPA.09, HIM.PA.154, CP.PMN.53). Uses lacking adequate documentation of efficacy and safety per those off‑label policies will be considered not authorized.

Coding

HCPCS CodesHCPCS

J0567

Injection, cerliponase alfa, 1 mg

inv-08: Maximum dose

Maximum dose300 mg every other week (maximum dosing for CLN2 therapy)

Administration routeIntraventricular infusion every other week

Approval duration related to dosingApproval duration: 6 months

inv-09: Minimum weight

Minimum weight requirement>= 2.5 kg

Where specifiedMember must weigh ≥ 2.5 kg to meet initial therapy criteria

Documentation noteWeight must be documented in provider submission for prior authorization

Provider Actions & Requirements

Prior Authorization

Prior Authorization Required

Prior authorization required — Provider must document that member meets all medical necessity criteria for Brineura, including diagnosis confirmation, neurologist involvement, weight requirement, and dosing limits. Providers must ensure evidence supporting each criterion is included with the request.

Approval duration: 6 months
Dose limit: ≤ 300 mg IV every other week
Prescriber: neurologist or in consultation with a neurologist

Note

Step Therapy

No step therapy requirements specified for Brineura in this policy. This is informational only — providers do not need to trial an alternative therapy prior to requesting Brineura under this policy.

Background

Neuronal ceroid lipofuscinosis type 2 (CLN2) is a pediatric, progressive neurodegenerative disorder caused by deficiency of the lysosomal enzyme tripeptidyl peptidase 1 (TPP1). TPP1 deficiency leads to accumulation of storage material in neurons, progressive loss of motor function and cognition, and eventual loss of ambulation. Cerliponase alfa (Brineura) is an intraventricular enzyme replacement therapy administered via an intraventricular access device to replace deficient TPP1 activity and slow the decline in motor function in patients with CLN2. Because therapy is delivered directly into the cerebrospinal fluid, careful attention to device‑related complications and infectious contraindications is necessary.

Definitions

inv-15: CLN2 Clinical Rating Scale (motor domain)

Score 3Walks normally

Score 2Intermittent falls, clumsiness, obvious instability

Score 1No unaided walking or crawling only

Score 0Immobile, mostly bedridden