CurrentUnitedHealthcarePolicy 2025D0065M

Brineura (cerliponase alfa) for CLN2 disease — Coverage Criteria

Defines UnitedHealthcare coverage criteria for Brineura (cerliponase alfa) to slow loss of ambulation in pediatric patients with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2/TPP1 deficiency); applies to clinical benefit determinations under UnitedHealthcare commercial medical benefit plans.

Policy Summary

PayerUnitedHealthcare

PolicyBrineura (cerliponase alfa) – coverage criteria for CLN2

Policy CodePolicy 2025D0065M

Change TypeRevised coverage languageadmin updates

Effective DateNov 1, 2025

Next Review Date

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

SourceLink

POLICY UPDATE CHANGES

Replaced language that tied proven status to meeting listed criteria with a statement that Brineura is proven for slowing loss of ambulation in pediatric CLN2 patients.

Updated FDA and References sections to reflect most current information.

300 mg q2wTherapeutic dosing (trials/guidance)

J0567HCPCS

E75.4Diagnosis

≤12 moInitial auth max

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Coverage Criteria for Brineura (cerliponase alfa)

Initial Therapy

Covered when ALL of the following are met:

Initial Coverage Criteria: Initial therapy is covered when all listed conditions are met

Diagnosis of Late Infantile Neuronal Ceroid Lipofuscinosis type 2 (CLN2) by a neurologist with expertise in the diagnosis of CLN2 OR diagnosis by a physician in consultation with a neurologist with expertise in the diagnosis of CLN2

Clinical score requirement: Combined motor + language score of 3 to 6 AND motor domain score of at least 1 AND language domain score of at least 1 on the CLN2 (LINCL) Clinical Scoring SystemCombined 3-6; motor ≥1; language ≥1

Prescribing clinician: Brineura is prescribed by a neurologist with expertise in the treatment of CLN2 OR by a physician in consultation with a neurologist with expertise in the treatment of CLN2

Administration and dosing: To be administered intraventricularly by, or under the direction of, healthcare professionals experienced in intraventricular infusions via an intracerebroventricular catheter; dosing in accordance with the FDA-approved labeling

Initial authorization will be for no more than 12 months

Continuation Therapy

Covered when ALL of the following are met:

Clinical response requirement: Patient has a score of 1 or higher in the motor domain of the CLN2 (LINCL) Clinical Scoring Systemmotor ≥1

Prescribing clinician: Brineura is prescribed by a neurologist with expertise in the treatment of CLN2 OR by a physician in consultation with a neurologist with expertise in the treatment of CLN2

Not Medically Necessary Indications

Excluded Indications: Brineura (cerliponase alfa) is unproven and not medically necessary for other forms of neuronal ceroid lipofuscinosis (non-CLN2 indications).

Brineura (cerliponase alfa) is unproven and not medically necessary for treatment of neuronal ceroid lipofuscinosis forms other than CLN2.

Use of cerliponase alfa for non-CLN2 forms of neuronal ceroid lipofuscinosis is considered not medically necessary and is excluded from coverage.

Coding and Dosing

Applicable HCPCSHCPCS

J0567

Injection, cerliponase alfa, 1 mg.

Applicable Diagnosis CodesICD-10

E75.4

Neuronal ceroid lipofuscinosis

Therapeutic dose in trials/guidelines

Therapeutic dose300 mg every 14 days (every other week)

Dose used in pivotal trialsDose-escalation to 300 mg every 14 days; maintained at 300 mg every 14 days in extension study

Guideline recommendationGuidelines suggest/recommend long-term ERT with cerliponase alfa 300 mg every other week (age-appropriate dosing permitted)

Route related dosing noteIntraventricular infusion dosing interval in trials and guidance is every 14 days; age-appropriate dose adjustments may apply per guidelines

Provider Actions, Authorization, and Documentation

Prior Authorization

Prior Authorization Required

Prior authorization is required before initiating Brineura (cerliponase alfa). Initial authorization will be for no more than 12 months; reauthorization (continuation) will be for no more than 12 months.

Prior authorization required
Initial authorization: ≤ 12 months
Reauthorization/continuation: ≤ 12 months

Documentation Required

Required Clinical Documentation

Providers must document diagnostic confirmation of CLN2 and involvement of a neurologist. Ensure documentation includes Clinical Scoring System for LINCL motor and language domain scores, prescriber specialty or documentation of consultation with a neurologist, and that dosing and administration will follow FDA labeling and be performed by clinicians experienced with intraventricular infusions via an intracerebroventricular catheter.

Background: CLN2 Disease and Therapy Context

Neuronal ceroid lipofuscinosis type 2 (CLN2) is an autosomal recessive pediatric neurodegenerative lysosomal storage disorder caused by deficiency of the lysosomal enzyme tripeptidyl peptidase 1 (TPP1). Affected children typically experience progressive motor decline, seizures, loss of vision, and premature death.

Cerliponase alfa (Brineura) is a recombinant proenzyme replacement therapy administered intraventricularly that is taken up via mannose-6-phosphate–mediated pathways and activated in lysosomes to replace deficient TPP1 activity.

Clinical trial evidence demonstrates that cerliponase alfa can slow decline in motor-language function and delay loss of ambulation in pediatric patients with CLN2; policy coverage and treatment criteria reference the CLN2 Clinical Scoring System motor and language domain scores to guide patient selection and continuation decisions.

Definitions

Cerliponase alfa

Generic and trade nameCerliponase alfa (Brineura)

Agent typeRecombinant proenzyme (rhTPP1) replacement therapy

Route of administrationIntraventricular infusion via intracerebroventricular catheter

Mechanism of actionTaken up by CNS cells via mannose-6-phosphate receptor, translocated to lysosomes where it is activated to cleave N-terminal tripeptides, replacing deficient TPP1 activity

CLN2 Clinical Scoring System (motor and language domains)

Purpose of the scaleQuantitative tool to describe clinical course and to rate motor and language function in CLN2 disease

Policy Summary

PayerUnitedHealthcare

PolicyBrineura (cerliponase alfa) – coverage criteria for CLN2

Policy CodePolicy 2025D0065M

Change TypeRevised coverage languageadmin updates

Effective DateNov 1, 2025

Next Review Date

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

SourceLink

On This Page

Reauthorization will be for no more than 12 months

Diagnosis of CLN2 documented by a neurologist with expertise in CLN2, or by a physician in consultation with such a neurologist
For initial therapy: combined motor + language domain score = 3–6, motor domain ≥1, language domain ≥1
For continuation therapy: motor domain score ≥1
Documentation that Brineura is prescribed by or in consultation with a neurologist experienced in CLN2 treatment
Documentation that administration will be intraventricular via intracerebroventricular catheter by or under direction of experienced healthcare professionals
Dosing consistent with FDA labeling

Denial Risk

Excluded Indication / Denial Risk

Brineura (cerliponase alfa) is considered unproven and not medically necessary for forms of neuronal ceroid lipofuscinosis other than CLN2 (late infantile neuronal ceroid lipofuscinosis type 2). Requests for Brineura for other NCL forms may be denied as not medically necessary.

Brineura is unproven/not medically necessary for other forms of neuronal ceroid lipofuscinosis (non-CLN2)

Note

Provider Operational Actions

Operational/provider directive: ensure all required documentation is submitted with the prior authorization request, including diagnostic confirmation, CLN2 Clinical Scoring System results, neurologist involvement or consultation notes, and plan for intraventricular administration by experienced clinicians.

Include relevant HCPCS (e.g., J0567) and diagnosis code (e.g., E75.4) on the request as applicable
Attach scoring documentation and specialist notes to avoid delays or denials

Scores motor function; policy requires motor domain score ≥1 for initial therapy and ≥1 for continuation (continuation specifically requires motor ≥1)

Language domainScores expressive language function; policy requires language domain score ≥1 and combined motor+language score 3–6 for initial therapy

Combined score relevanceInitial therapy criteria require combined motor + language score between 3 and 6 inclusive