CurrentUnitedHealthcarePolicy CS2025D0065Q

Brineura (Cerliponase Alfa) — Medical Benefit Drug Policy

This policy governs medical benefit coverage criteria for Brineura (cerliponase alfa) for treatment of late infantile neuronal ceroid lipofuscinosis type 2 (CLN2/TPP1 deficiency) for UnitedHealthcare Community Plan members, excluding certain states with separate guidance.

Policy Summary

PayerUnitedHealthcare

PolicyBrineura (Cerliponase Alfa) — Medical Benefit Drug Policy

Policy CodePolicy CS2025D0065Q

Change TypeNo material change

Effective DateNov 1, 2025

Next Review Date

Key ActionObtain prior authorization; initial and reauthorization approvals are limited to no more than 12 months when coverage criteria are met.

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

300 mg q2wFDA dosing

12 monthsInitial auth ≤

12 monthsReauth ≤

J0567Billing code

E75.4Diagnosis code

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inv-01: Initial therapy — Covered when ALL of the following are met

Covered when ALL of the following are met

Initial coverage criteria: All of the following must be met:

Diagnosis of late infantile neuronal ceroid lipofuscinosis type 2 (CLN2) by a neurologist with expertise in the diagnosis of CLN2 OR diagnosis of CLN2 by a physician in consultation with a neurologist with expertise in the diagnosis of CLN2.

Clinical severity (CLN2 Clinical Scoring System): Combined motor and language domain score of 3 to 6 on the CLN2 Clinical Scoring System AND score of at least 1 in the motor domain AND score of at least 1 in the language domain.Combined motor+language 3-6; motor >=1; language >=1

Prescriber, administration, dosing, and authorization: Brineura to be administered intraventricularly by, or under the direction of, healthcare professionals experienced in intraventricular infusions via an intracerebroventricular catheter; dosing in accordance with FDA‑approved labeling; initial authorization for no more than 12 months.

inv-02: Continuation therapy — Covered when ALL of the following are met

Covered when ALL of the following are met

Continuation coverage criteria: All of the following must be met:

Clinical status: Patient has a score of 1 or higher in the motor domain of the CLN2 Clinical Scoring System.motor >=1

Prescriber: Brineura prescribed by a neurologist with expertise in the treatment of CLN2 OR prescribed by a physician in consultation with a neurologist with expertise in the treatment of CLN2.

inv-03: Not medically necessary / Unproven

Other forms of neuronal ceroid lipofuscinosis: Brineura is unproven and not medically necessary for other forms of neuronal ceroid lipofuscinosis (non-CLN2).

Use of Brineura (cerliponase alfa) for forms of neuronal ceroid lipofuscinosis other than late infantile neuronal ceroid lipofuscinosis type 2 (CLN2) is considered unproven and not medically necessary.

Brineura (cerliponase alfa) is unproven and not medically necessary for treatment of neuronal ceroid lipofuscinosis when the diagnosis is not CLN2 (TPP1 deficiency).

Provider Actions, Authorization, and Documentation

Prior Authorization

Prior Authorization Required

Prior authorization is required. Initial approvals are limited to no more than 12 months; reauthorization (continuation) approvals are limited to no more than 12 months.

Initial and reauthorization periods: ≤ 12 months

Step Therapy

No step therapy is specified for this policy.

Documentation Required

Required Clinical Documentation

Documentation must support the diagnosis of late infantile neuronal ceroid lipofuscinosis type 2 (CLN2) by a neurologist with expertise in the diagnosis of CLN2, or by a physician in consultation with such a neurologist. For initial therapy, documentation must include Clinical Scoring System for LINCL: combined motor and language score of 3–6, with at least 1 in the motor domain and at least 1 in the language domain. For continuation therapy, documentation must show a motor domain score of ≥1. Also document that Brineura will be administered intraventricularly by or under the direction of clinicians experienced with intracerebroventricular catheter infusions, and that dosing follows FDA‑approved labeling.

Diagnosis must be by a neurologist with CLN2 expertise or a physician in consultation with such a neurologist
Initial Clinical Scoring System for LINCL: combined motor+language = 3–6; motor ≥1; language ≥1
Continuation: motor domain score ≥1
Administration: intraventricular via intracerebroventricular catheter by experienced clinicians
Dosing per FDA labeling

Denial Risk

Documentation Deficiencies May Result in Denial

Lack of documentation meeting initial or continuation criteria — including absence of the required specialist diagnosis/consultation, missing Clinical Scoring System scores (initial: combined 3–6 with motor ≥1 and language ≥1; continuation: motor ≥1), or failure to document appropriate intraventricular administration and FDA‑label dosing — may result in claim denial or request for additional information.

Missing neurologist diagnosis or documented consultation
Missing required LINCL Clinical Scoring System scores
No documentation of intraventricular administration by experienced clinicians
No documentation that dosing follows FDA labeling

Background

Late infantile neuronal ceroid lipofuscinosis type 2 (CLN2), caused by deficient tripeptidyl peptidase‑1 (TPP1) activity, is a pediatric autosomal recessive neurodegenerative lysosomal storage disorder characterized by progressive motor decline, seizures, language impairment and vision loss. Cerliponase alfa (Brineura) is an intraventricular enzyme replacement administered via an intracerebroventricular catheter to deliver functional TPP1 to the central nervous system and is indicated to slow loss of ambulation in patients with CLN2; dosing must follow the U.S. Food and Drug Administration labeling and administration must be performed by or under the direction of clinicians experienced with intracerebroventricular infusions. Initial and reauthorization approval periods are limited to no more than 12 months when coverage criteria are met.

Definitions and Scales

inv-14: CLN2 (TPP1 deficiency) — Late infantile neuronal ceroid lipofuscinosis type 2, a childhood-onset neurodegenerative lysosomal storage disorder caused by deficient ...

Condition nameLate infantile neuronal ceroid lipofuscinosis type 2 (CLN2) — tripeptidyl peptidase‑1 (TPP1) deficiency

PathophysiologyDeficient TPP1 enzyme activity leading to accumulation of lysosomal storage material in the CNS

Clinical featuresPediatric‑onset progressive motor decline, seizures, language delay, movement disorders, vision loss, dementia, early death

GeneticsAutosomal recessive inheritance

inv-15: CLN2 Clinical Scoring System (motor and language domains) — A quantitative rating scale for CLN2 measuring motor and language function; used to determine eligibility and monitor response.

PurposeQuantitative rating scale for CLN2 to describe clinical course and measure motor and language function

Domains usedMotor and language domains

Eligibility thresholds (per policy)Combined motor+language score 3–6 with motor ≥1 and language ≥1 for initial coverage; motor ≥1 required for continuation

UseUsed to determine eligibility for treatment and to monitor response over time