CurrentColorado Rocky Mountain Health PlansPolicy CS2026D0065R

Brineura (Cerliponase Alfa)

Defines medical benefit coverage criteria for Brineura (cerliponase alfa) for pediatric patients with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2/TPP1 deficiency), including initial and continuation authorization, administration requirements, dosing alignment with FDA labeling, and applicable HCPCS/diagnosis codes. Excludes certain state-specific applicability noted in the Application section.

Policy Summary

PayerColorado Rocky Mountain Health Plans

PolicyBrineura (Cerliponase Alfa)

Policy CodePolicy CS2026D0065R

Change TypeRevised application language (state applicability)

Effective DateApr 1, 2026

Next Review Date

Key ActionSubmit initial and reauthorization requests with documentation of diagnosis by or in consultation with a CLN2-experienced neurologist, CLN2 clinical scores, prescribing clinician specialty, intraventricular administration capability, and dosing per FDA label; authorizations are limited to no more than 12 months.

POLICY UPDATE CHANGES

Application: Removed language indicating this policy does not apply to the state of Indiana and removed content/language pertaining to the state of Louisiana.

1Primary HCPCS code listed

1Relevant ICD-10 diagnosis code listed

12 monthsMax authorization duration (initial/repeat)

3–6Required combined motor+language score (initial)

Coverage Summary

Scope: Brineura (cerliponase alfa) is covered with criteria for pediatric late infantile neuronal ceroid lipofuscinosis type 2 (CLN2/TPP1 deficiency) per policy CS2026D0065R (effective April 1, 2026). Coverage requires documentation of diagnosis by or in consultation with a neurologist experienced in CLN2, fulfillment of the specified CLN2 clinical scoring thresholds for initial or continuation therapy, administration via an intracerebroventricular (intraventricular) catheter by or under the direction of clinicians experienced with intraventricular infusions, and dosing in accordance with the U.S. Food and Drug Administration (FDA) approved labeling (authorization limited to no more than 12 months for initial or reauthorization).

Initial Therapy Criteria

Initial Therapy

Covered when ALL of the following are met:

ALL of the following

Diagnosis by specialist
- 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
- Combined score of 3 to 6 in the motor and language domains of the Clinical Scoring System for LINCL3 to 6 (combined)
- Score of at least 1 in the motor domain of the Clinical Scoring System for LINCL>= 1 (motor)
- Score of at least 1 in the language domain of the Clinical Scoring System for LINCL>= 1 (language)
Prescribing clinician
- 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
Brineura is to be administered intraventricularly by, or under the direction of, healthcare professionals experienced in performing intraventricular infusions via an intracerebroventricular catheter
Dosing is in accordance with the U.S. Food and Drug Administration (FDA) approved labeling
Initial authorization will be for no more than 12 months

Prior Authorization

Initial prior authorization required

Initial authorization (up to 12 months) requires documentation that the patient has CLN2 diagnosed by or in consultation with a neurologist experienced in CLN2; combined motor+language CLN2 score 3–6 with motor ≥1 and language ≥1; the prescribing clinician is a neurologist experienced in CLN2 or a physician in consultation with such a neurologist; the therapy will be administered intraventricularly by or under the direction of clinicians experienced with intracerebroventricular catheter infusions; and dosing follows the FDA-approved labeling.

J0567
E75.4
Initial authorization up to 12 months

Continuation Therapy (Reauthorization) Criteria

Continuation of Therapy (Reauthorization)

Covered when ALL of the following are met:

ALL of the following

Diagnosis by specialist
- 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 LINCL>= 1 (motor)
Prescribing clinician

Not Medically Necessary / Exclusions

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

Applicable Codes

Covered HCPCSHCPCSCovered

J0567

Injection, cerliponase alfa, 1 mg

Relevant Diagnosis Codes (reference)ICD-10

E75.4

Neuronal ceroid lipofuscinosis

Provider Actions & Documentation Requirements

Documentation Required

Administration setting and clinician qualifications

Document that Brineura will be administered intraventricularly by, or under the direction of, healthcare professionals experienced in performing intraventricular infusions via an intracerebroventricular catheter, and that dosing will follow the FDA-approved labeling.

Provider actions and billing: Prior authorization criteria must be met and documented to support coverage and avoid denial of claims for HCPCS code J0567 and related services; documentation should include the CLN2 diagnosis (ICD-10 E75.4), specialist diagnosis/consultation, required CLN2 clinical scores, prescribing clinician specialty or consultation, intraventricular administration capability, and dosing per the FDA label.

Clinical Evidence Summary

<3 yrs: motor=3Treated children <3 years maintained motor score of 3 (Study 190-203)

Evidence summary: Clinical trials and extension studies demonstrated that intraventricular cerliponase alfa slowed decline in motor and language (motor-language) scores versus historical controls and produced a high response rate in phase 1/2 and extension data. In the phase 1/2 study and extension, 87% of treated patients met the primary endpoint (absence of an unreversed two-point decline or score of zero at Week 48) versus an expected 50% (p = 0.0002). In the multicenter open-label trial (Schulz et al.), the mean unadjusted rate of decline per 48 weeks was 0.27 ± 0.35 points for treated patients versus 2.12 ± 0.98 points for 42 historical controls (mean difference, 1.85; p < 0.001), and median time to a two-point decline was not reached in treated patients versus 345 days in historical controls. Study 190-203 showed treated children <3 years maintained a motor score of 3 while matched natural history comparators had a 61% rate of unreversed two-point decline or score of zero by final assessment, supporting slowed motor decline with intraventricular administration at FDA-recommended dosing every 2 weeks.

Background & Definitions

Background: Brineura (cerliponase alfa) is indicated to slow the loss of ambulation in pediatric patients with CLN2 (TPP1 deficiency). It is an intraventricular enzyme replacement therapy (a proenzyme taken up into CNS cells and targeted to lysosomes) that must be administered via an intracerebroventricular catheter. Clinical studies and guideline recommendations describe dosing as every 2 weeks (with 300 mg or age-appropriate dosing maintained in extension studies) delivered by intraventricular infusion.

Definitions: CLN2 Clinical Rating Scale — a scale measuring the motor and language domains used to quantify clinical course over time; the motor domain measures ambulation where 3 = normal and 0 = no function. Decline is defined as a sustained two-point loss or a score of zero (irreversible) in the scale's domain assessments.

Revision History

2026-04-01revisedLatest

Application: Removed language indicating this policy does not apply to the state of Indiana and removed content/language pertaining to the state of Louisiana. Supporting Information: Archived previous policy versions CS2025D0065Q and CSIND0065.07.