RetiredUnitedHealthcarePolicy CSLA2026D0065M

Brineura (cerliponase alfa) (for Louisiana Only) Retired April 1, 2026

UnitedHealthcare Medical Benefit Drug Policy for Brineura (cerliponase alfa) applicable to Louisiana membership, defining initial and continuation authorization clinical criteria, coding references, and policy status (retired effective April 1, 2026).

Policy Summary

PayerUnitedHealthcare

PolicyBrineura (cerliponase alfa) (for Louisiana Only) Retired April 1, 2026

Policy CodePolicy CSLA2026D0065M

Change TypeRetired

Effective DateJan 1, 2026

Next Review Date

Key ActionProviders must document diagnosis by or in consultation with a neurologist experienced in CLN2 and provide CLN2 motor and language scores to support authorization; approvals limited to no more than 12 months and dosing/administration must follow FDA labeling.

POLICY UPDATE CHANGES

Policy retired effective April 1, 2026; Louisiana plan membership disenrolled on Apr. 1, 2026.

Updated FDA and References sections as of 01/01/2026.

1HCPCS drug code listed

1Diagnosis code listed

12 moMax auth (initial/renewal)

RetiredPolicy status

Coverage Summary

This policy addresses Brineura (cerliponase alfa) for CLN2 (tripeptidyl peptidase 1 [TPP1] deficiency) applicable to Louisiana membership. Coverage stance is mixed — the drug is covered when specific clinical criteria are met and is not covered for other forms of neuronal ceroid lipofuscinosis. The policy subject is Brineura (cerliponase alfa) for CLN2 (Louisiana only). Policy status is RETIRED — the policy was retired effective April 1, 2026 (effective period shown January 1, 2026 – March 31, 2026) and Louisiana plan membership was disenrolled on April 1, 2026.

Key thresholds and policy status

Combined motor+language CLN2 score (initial)3 to 6

Motor domain CLN2 score (initial)>= 1

Language domain CLN2 score (initial)>= 1

Motor domain CLN2 score (continuation)>= 1

Max authorization duration (initial/renewal)12 months

Policy statusRetired 2026-04-01

HCPCS drug code listed1 (J0567)

Diagnosis code listed1 (E75.4)

Initial Therapy Criteria (Medical Necessity)

Initial Therapy - Medical Necessity Criteria

Covered when ALL of the following are met:

ALL of the following

Diagnosis established 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 domains3 to 6
- Score of at least 1 in the motor domain>= 1
- Score of at least 1 in the language domain>= 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
Brineura is to be administered intraventricularly by, or under the direction of, healthcare professionals experienced in performing intraventricular infusions via an intracerebroventricular catheter
administration competence requirement
Dosing is in accordance with the United States Food and Drug Administration approved labeling
must follow FDA labeling
Initial authorization will be for no more than 12 months12 months

Continuation Therapy Criteria (Reauthorization)

Continuation Therapy - Medical Necessity Criteria

Reauthorization covered when ALL of the following are met:

ALL of the following

Diagnosis established 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 LINCL>= 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
Brineura is to be administered intraventricularly by, or under the direction of, healthcare professionals experienced in performing intraventricular infusions via an intracerebroventricular catheter
administration competence requirement
Dosing is in accordance with the United States Food and Drug Administration approved labeling
must follow FDA labeling
Reauthorization will be for no more than 12 months12 months

Not Medically Necessary / Not Covered

Not covered when the following condition applies:

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

Provider Actions & Documentation Requirements

Prior Authorization

Authorization required for initial and continuation therapy

Initial and reauthorization approvals are limited to no more than 12 months each and require meeting the policy clinical criteria: diagnosis established by/with a neurologist experienced in CLN2, required CLN2 Clinical Scoring System motor and language domain scores, intraventricular administration via intracerebroventricular catheter by qualified professionals, and dosing per FDA labeling.

J0567
E75.4

Documentation Required

Clinical documentation of diagnosis and CLN2 scores

Document that the diagnosis of CLN2 was established by a neurologist with expertise in CLN2 or by a physician in consultation with such a neurologist, and provide motor and language domain scores from the CLN2 Clinical Scoring System to support authorization requests.

CLN2 Clinical Scoring System motor and language domain scores

Billing Rule

Administration route requirement

Cerliponase alfa (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 the appropriate administration and the HCPCS J-code for cerliponase alfa.

J0567

Clinical Evidence Summary

87%Phase 1/2 & extension responders at 48 weeks (20/23) vs expected 50%; p=0.0002

0%Study 190-203: treated <3 yrs had 0% unreversed two-point motor decline or score of 0 by week 169

1.85Schulz et al.: mean motor-language decline difference per 48 weeks (treated 0.27 vs controls 2.12); p<0.001

Brineura is an intraventricular enzyme replacement therapy indicated to slow the loss of ambulation in pediatric patients with CLN2 (TPP1 deficiency). It is administered via intraventricular infusion using an intracerebroventricular catheter and dosed in accordance with FDA-approved labeling.

The CLN2 Clinical Scoring System (LINCL) is a clinical rating scale that measures motor and language domains to quantify disease progression in late infantile neuronal ceroid lipofuscinosis and is used to determine eligibility and treatment response.

An intraventricular infusion / intracerebroventricular catheter refers to the route and device required for administration of cerliponase alfa into the ventricular system of the brain; Brineura must be administered intraventricularly by, or under the direction of, healthcare professionals experienced in performing intraventricular infusions via an intracerebroventricular catheter.

Background & Definitions

Brineura (cerliponase alfa) is indicated to slow the loss of ambulation in children with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2), a pediatric autosomal recessive neurodegenerative lysosomal storage disorder caused by deficient tripeptidyl peptidase-1 (TPP1) activity. CLN2 leads to accumulation of lysosomal storage material in the central nervous system, progressive motor decline, seizures, language delay, movement disorders, vision loss, and early death.

Clinical evidence from an open-label multicenter study and phase 1/2 trials demonstrates that intraventricular infusion of cerliponase alfa every two weeks slows decline in motor and language function compared with historical controls, with primary and secondary analyses showing reduced rate of motor-language score decline and high response rates at 48 weeks.

Studies used the CLN2 Clinical Rating Scale (motor and language domains) to assess outcomes; treatment is given by intraventricular infusion via an intracerebroventricular catheter and dosing follows FDA-approved labeling.

CLN2 Clinical Scoring System (LINCL): A clinical rating scale measuring motor and language domains used to quantify disease progression in late infantile neuronal ceroid lipofuscinosis and to establish eligibility and monitor response to therapy.

Intraventricular infusion / intracerebroventricular catheter: The route and device required for administration of cerliponase alfa into the ventricular system of the brain; Brineura must be administered intraventricularly by, or under the direction of, healthcare professionals experienced in performing intraventricular infusions via an intracerebroventricular catheter.

Revision History

2026-04-01retirementLatest

Policy retired effective April 1, 2026; Louisiana plan membership disenrolled on Apr. 1, 2026. Retirement noted in 04/01/2026 summary of changes; prior version archived.

2026-01-01supporting_information_update

Supporting information updated; FDA and References sections revised and previous policy version CSLA2025D0065L archived.