CurrentUnitedHealthcarePolicy IEXD0065.09

Brineura® (Cerliponase Alfa) – Individual Exchange Medical Benefit Drug Policy

Defines UnitedHealthcare Individual Exchange medical benefit drug policy for Brineura (cerliponase alfa) for pediatric patients with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2/TPP1 deficiency), including initial and continuation authorization criteria, applicable HCPCS/diagnosis codes, dosing/admin requirements, and authorization durations. Applies to Individual Exchange plans in all states except MA, NV, and NY.

Policy Summary

PayerUnitedHealthcare

PolicyBrineura® (Cerliponase Alfa) – Individual Exchange Medical Benefit Drug Policy

Policy CodePolicy IEXD0065.09

Change TypeSupporting information update (FDA & References); archived prior version

Effective DateNovember 1, 2025

Next Review Date

Key ActionSubmit authorization requests documenting diagnostic confirmation, CLN2 clinical scoring per policy, prescriber expertise/consultation, intraventricular administration by experienced clinicians, and dosing per FDA label; approvals limited to a maximum of 12 months.

SourceLink

POLICY UPDATE CHANGES

Updated FDA and References sections; archived previous policy version IEXD0065.08.

1Covered Indication (CLN2)

1HCPCS listed (J0567)

1ICD-10 listed (E75.4)

Coverage Summary

Defines UnitedHealthcare Individual Exchange medical benefit drug policy for Brineura (cerliponase alfa) for pediatric patients with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2/TPP1 deficiency). Applies to Individual Exchange plans in all states except MA, NV, and NY. Coverage stance: covered_with_criteria. Policy addresses initial and continuation authorization criteria, applicable HCPCS and diagnosis codes, dosing and administration requirements, and authorization durations. Initial authorization is for no more than 12 months, and reauthorization (continuation) is for no more than 12 months.

Authorization thresholds

Combined motor and language score3 to 6

Motor domain score (initial)>= 1

Language domain score (initial)>= 1

Motor domain score (continuation)>= 1

Initial Therapy — Medically Necessary Criteria

Initial therapy — medically necessary criteria

Brineura is proven and medically necessary when ALL of the following are met:

ALL of the following

Diagnosis by
- Diagnosis of late infantile neuronal ceroid lipofuscinosis type 2 (CLN2) by a neurologist with expertise in the diagnosis of CLN2
- Diagnosis of late infantile neuronal ceroid lipofuscinosis type 2 (CLN2) by a physician in consultation with a neurologist with expertise in the diagnosis of CLN2
Clinical scoring requirements (Clinical Scoring System for LINCL)
- Combined score of 3 to 6 in the motor and language domainsCombined motor and language score: 3 to 6
- Score of at least 1 in the motor domainMotor domain score (initial): >= 1
- Score of at least 1 in the language domainLanguage domain score (initial): >= 1
Prescriber
- Brineura is prescribed by a neurologist with expertise in the treatment of CLN2
- Brineura is prescribed by a physician in consultation with a neurologist with expertise in the treatment of CLN2
Administration and dosing: Brineura is to be administered intraventricularly by, or under the direction of, healthcare professionals experienced in performing intraventricular infusions via an intracerebroventricular catheter
administration route/setting requirement
Dosing is in accordance with the United States Food and Drug Administration approved labeling
dose must follow FDA label
Authorization duration: Initial authorization will be for no more than 12 months<= 12 months

Continuation (Reauthorization) — Medically Necessary Criteria

Continuation (reauthorization) therapy — medically necessary criteria

Reauthorization is permitted when ALL of the following are met:

ALL of the following

Diagnosis by
- Diagnosis of late infantile neuronal ceroid lipofuscinosis type 2 (CLN2) by a neurologist with expertise in the diagnosis of CLN2
- Diagnosis of late infantile neuronal ceroid lipofuscinosis type 2 (CLN2) by a physician in consultation with a neurologist with expertise in the diagnosis of CLN2
Patient has a score of 1 or higher in the motor domain of the Clinical Scoring System for LINCLMotor domain score (continuation): >= 1
Prescriber
- Brineura is prescribed by a neurologist with expertise in the treatment of CLN2
- Brineura is prescribed by a physician in consultation with a neurologist with expertise in the treatment of CLN2
Administration and dosing: Brineura is to be administered intraventricularly by, or under the direction of, healthcare professionals experienced in performing intraventricular infusions via an intracerebroventricular catheter
administration route/setting requirement
Dosing is in accordance with the United States Food and Drug Administration approved labeling
dose must follow FDA label
Authorization duration: Reauthorization will be for no more than 12 months<= 12 months

Provider Actions & Billing

Prior Authorization

Authorization required

Initial and reauthorization approvals limited to a maximum of 12 months and require documentation of diagnostic confirmation, CLN2 clinical scoring per policy, prescriber expertise/consultation, intraventricular administration by experienced clinicians, and dosing per FDA label.

J0567
E75.4

Documentation Required

Clinical scoring documentation

Document the Clinical Scoring System for LINCL motor and language domain scores as part of the authorization request. For initial authorization, document a combined motor+language score of 3 to 6 with each component (motor and language) ≥ 1. For continuation/reauthorization, document motor domain score ≥ 1.

Combined motor+language score 3 to 6; motor ≥ 1; language ≥ 1 (initial)
Motor ≥ 1 (continuation)

Billing Rule

Admin route requirement

Brineura must be administered intraventricularly via an intracerebroventricular catheter by, or under the direction of, healthcare professionals experienced in performing intraventricular infusions. Billing should reflect HCPCS J0567 (injection, cerliponase alfa, 1 mg) for drug supply per mg.

J0567

Clinical Evidence

87%Phase 1/2 and extension response rate at Week 48 (absence of unreversed two-point decline or score of zero)

0.27 vs 2.12NEJM study: mean unadjusted rate of decline per 48 weeks — 0.27 ±0.35 treated vs 2.12 ±0.98 controls (mean difference 1.85; p < 0.001)

Cerliponase alfa (Brineura) is an intraventricular enzyme replacement therapy for CLN2 caused by TPP1 deficiency; trials showed it slowed decline in motor and language function versus historical controls. In pivotal studies and extension trials dosing was 300 mg every 2 weeks administered intraventricularly.

Background & Definitions

Cerliponase alfa (Brineura) is a proenzyme taken up by CNS target cells and translocated to lysosomes via the CI-MPR (M6P/IGF2) receptor; it is activated in lysosomes where the proteolytic form cleaves tripeptides from the N-terminus of proteins. It is indicated to slow loss of ambulation in pediatric patients with CLN2 (TPP1 deficiency). Pivotal dosing context: intraventricular infusion with doses up to 300 mg every 14 days (maintained at 300 mg every 14 days in extension studies) and efficacy assessed by the CLN2 Clinical Rating Scale comparing treated patients to historical controls.

CLN2 Clinical Rating Scale (Clinical Scoring System for Late Infantile Neuronal Ceroid Lipofuscinoses) is used to assess motor and language domains; the motor domain measures ambulation (3 = normal, 0 = no function).

Revision History

2025-11-01policy_effectiveLatest

Effective date: Policy IEXD0065.09 became effective.

2025-11-01last_reviewLatest

Last review date: 2025-11-01.

2025-11-01archived_previous_versionLatest

Archived previous policy version IEXD0065.08; Updated FDA and References sections to reflect the most current information.