CurrentUnitedHealthcarePolicy CSOH2026D0065.D

Brineura Cerliponase Alfa Oh Cs

State-specific UnitedHealthcare Medical Benefit Drug Policy for Brineura (cerliponase alfa) applicable only to Ohio; defines medical necessity criteria for initiation and continuation of therapy, coding references, and administrative notes. It references FDA labeling, dosing per FDA, and requires administration via intraventricular infusion by experienced providers.

Policy Summary

PayerUnitedHealthcare

PolicyBrineura Cerliponase Alfa Oh Cs

Policy CodePolicy CSOH2026D0065.D

Change TypeUpdated FDA and References

Effective DateJan 1, 2026

Next Review Date

Key ActionInitial and reauthorization requests must meet the specified diagnostic and Clinical Scoring System criteria; initial and renewals are authorized for up to 12 months.

SourceLink

POLICY UPDATE CHANGES

Updated FDA and References sections to reflect the most current information (01/01/2026).

1HCPCS drug code listed

1Diagnosis code listed

12 monthsMax authorization duration

Combined score 3-6Initial therapy clinical score band

Coverage Summary

Coverage stance: covered_with_criteria under a State-specific UnitedHealthcare Medical Benefit Drug Policy applicable to Ohio only. This policy (CSOH2026D0065.D, effective 01/01/2026) defines medical necessity criteria for Brineura (cerliponase alfa).

Indication: Brineura is indicated to slow the loss of ambulation in pediatric patients with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2; TPP1 deficiency).

Dosing and administration: Dosing and administration are per the FDA-approved labeling (for example, intraventricular infusion with a common dosing regimen of 300 mg every 2 weeks).

Authorization duration limit: Initial and reauthorization approvals are each limited to no more than 12 months.

Key thresholds and quick facts

Combined motor + language score (eligibility)3 to 6 on the CLN2 (LINCL) Clinical Scoring System

Motor domain score (eligibility)Score of at least 1 on the motor domain

Language domain score (eligibility)Score of at least 1 on the language domain

Maximum authorization durationInitial and reauthorization approvals up to 12 months

HCPCS drug codeJ0567 — Injection, cerliponase alfa, 1 mg

Diagnosis code (reference)E75.4 — Neuronal ceroid lipofuscinosis (CLN2)

Initial Therapy - Medically Necessary Criteria

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

ALL of the following

Diagnosis confirmation
- 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
- Combined score of 3 to 6 in the motor and language domains of the Clinical Scoring System for LINCLcombined score 3-6
- Score of at least 1 in the motor domain of the Clinical Scoring System for LINCLmotor domain >= 1
- Score of at least 1 in the language domain of the Clinical Scoring System for LINCLlanguage domain >= 1
Prescriber requirement
- 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
  Operational requirement for administration setting/providers
- Dosing is in accordance with the U.S. Food and Drug Administration (FDA) approved labeling
  Follow FDA-approved dosing (e.g., 300 mg every 2 weeks per label where applicable)
Authorization duration: Initial authorization will be for no more than 12 months
Authorization duration limit

Continuation Therapy - Medically Necessary Criteria

For reauthorization, ALL of the following must be met:

ALL of the following

Diagnosis confirmation
- 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 (continuation): Patient has a score of 1 or higher in the motor domain of the Clinical Scoring System for LINCLmotor domain >= 1
Prescriber requirement
- 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 provider requirement
- Dosing is in accordance with the U.S. Food and Drug Administration (FDA) approved labeling
  Follow FDA-approved dosing
Authorization duration: Reauthorization will be for no more than 12 months
Authorization duration limit

Provider Actions / Prior Authorization

Prior Authorization

Prior authorization required for initial and continuation therapy

Initial and reauthorization requests must meet the specified diagnostic and Clinical Scoring System criteria and are authorized for up to 12 months.

J0567

Documentation Required

Prescriber and diagnosis documentation

Documentation must show diagnosis of CLN2 by a neurologist with expertise or a physician in consultation with such a neurologist, and must include the CLN2 (Clinical Scoring System for LINCL) motor and language domain scores required for initial or continuation coverage.

Billing Rule

Administration route and provider capability

Claims should reflect intraventricular administration, and the provider performing administration must be experienced in intraventricular infusions via an intracerebroventricular catheter.

J0567

Clinical Evidence

Slowed declineslowed decline in motor function (Study 190-203)

Includes <3 yrsdelayed disease onset including children <3 years

Brineura is an intraventricular enzyme replacement therapy for CLN2. Clinical trials (including a multicenter open-label study and phase 1/2 studies with extension) demonstrated slowed decline in motor-language scores versus historical controls and an 87% response rate at Week 48 by the protocol-defined endpoint. Dosing is per the FDA label, commonly 300 mg every 2 weeks.

Background & Definitions

Brineura (cerliponase alfa) is an enzyme replacement therapy administered intraventricularly to slow loss of ambulation in pediatric CLN2 (TPP1 deficiency). It is delivered via intraventricular infusion through an intracerebroventricular catheter. Clinical studies evaluated dosing from 30 to 300 mg every 14 days and the maintenance dose in extension studies was 300 mg every 2 weeks.

Clinical Scoring System for LINCL (CLN2 Clinical Rating Scale): a scale measuring the motor and language domains used to quantify clinical course over time. The motor domain assesses ambulation with scores from 3 (normal) to 0 (no function). In study definitions, decline is defined as a sustained two-point loss or a score of zero (unreversed).