CurrentBaylor Scott & White Health PlanPolicy 238

Cerliponase alfa (Brineura) for CLN2 disease

This policy governs prior authorization and medical necessity requirements for use of cerliponase alfa (Brineura) to treat neuronal ceroid lipofuscinosis type 2 (CLN2) for Baylor Scott & White Health Plan members.

Policy Summary

PayerBaylor Scott & White Health Plan

PolicyCerliponase alfa (Brineura) for CLN2 disease

Policy CodePolicy 238

Change TypeRemoved age criteriondocumentation requirement added

Effective DateNov 1, 2025

Next Review DateSep 8, 2026

Key ActionSubmit prior authorization with documentation showing CLN2 diagnosis (TPP1 deficiency or biallelic pathogenic TPP1 variants) and CLN2 CRS motor and language scores.

SourceLink

POLICY UPDATE CHANGES

Added requirement that documentation must be submitted with requests.

Universal and renewal request headings updated and standardized language for indication, dosing, and experimental/investigational language.

Removed age criteria consistent with expanded FDA indication (policy now reflects broader age coverage).

Prior authutilization control

Diagnosis CLN2required documented diagnosis (E75.4 may apply)

J0567drug billing code listed

Efficacy requiredrenewal criterion

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Coverage Criteria for Cerliponase alfa (Brineura)

Initial Therapy

Covered when ALL of the following are met:

Initial coverage criteria: 1) Diagnosis of neuronal ceroid lipofuscinosis type 2 (CLN2) documented by EITHER: a) deficiency of tripeptidyl peptidase 1 (TPP1) in leukocytes or fibroblasts, OR b) demonstration of biallelic pathogenic or likely pathogenic variants in the TPP1 gene; 2) Combined score of 3–6 on the motor and language domains of the CLN2 clinical rating scale (CRS) with at least 1 point in each domain; 3) Cerliponase alfa will be administered by, or under the direction of, a physician knowledgeable in intraventricular administration and management of hypersensitivities (e.g., anaphylaxis); 4) Cerliponase alfa will be dosed and administered according to FDA-approved labeling; 5) Member does NOT have any of the following exclusions: a) age < 37 weeks post‑menstrual age; b) weight < 2.5 kg; c) sign or symptom of acute or unresolved localized infection at or around the device insertion site (e.g., cellulitis or abscess); d) suspected or confirmed CNS infection (e.g., cloudy CSF, positive CSF gram stain, meningitis); e) acute intraventricular access device‑related complications (leakage, device failure, extravasation); f) presence of a ventriculoperitoneal shunt; g) other FDA‑labeled contraindications to cerliponase alfa.

Renewal Therapy

Renewal is covered when ALL of the following are met:

Renewal criteria: 1) Member continues to meet all universal/initial criteria (see Initial Therapy); 2) Documentation of efficacy evidenced by either: a) no loss of ambulation, OR b) slowed loss of ambulation compared with baseline; 3) Adverse effects are manageable.

Other Indications (Not Covered)

Not covered indications: Use of cerliponase alfa (Brineura) for all indications other than those specified in this policy is considered experimental and investigational (not medically necessary) because effectiveness has not been established.

Exclusion criteria: Cerliponase alfa is not covered when any of the following apply. Member is 37 weeks post‑menstrual age (gestational age at birth plus post‑natal age); OR member weighs 2.5 kg; OR there is any sign or symptom of an acute or unresolved localized infection at or around the intraventricular device insertion site (for example, cellulitis or abscess); OR suspected or confirmed central nervous system infection (for example, cloudy CSF, positive CSF gram stain, or meningitis); OR acute intraventricular access device–related complications (such as leakage, device failure, or extravasation of fluid); OR the presence of a ventriculoperitoneal shunt; OR any other FDA‑labeled contraindication to cerliponase alfa.

Use of cerliponase alfa (Brineura) for any indications other than those specified in this policy is considered experimental and investigational (not medically necessary) because effectiveness has not been established. All such requests will be reviewed by a clinical pharmacist and a medical director.

Coding and Clinical Scales

HCPCS CodesHCPCSCovered

J0567

Injection, cerliponase alfa, 1 mg.

ICD-10 Codes (may apply)ICD-10

E75.4

Neuronal ceroid lipofuscinosis

CLN2 clinical rating scale (motor + language) — combined score requirements

Combined CLN2 CRS scoreCombined motor + language score of 3–6

Minimum per-domain scoreAt least 1 point in each of the motor and language domains

Score use contextRequired as part of universal criteria for coverage requests

Provider Actions, Documentation, and Authorization

Prior Authorization

Prior authorization required; clinical review

Prior authorization is required for cerliponase alfa (Brineura). Requests must document the diagnostic and clinical criteria described in the policy and will be reviewed by a clinical pharmacist and a medical director.

Relevant billing codes: J0567; diagnosis code E75.4 may apply.

Step Therapy

Step therapy not specified

No step therapy requirements apply for cerliponase alfa under this policy.

Documentation Required

Required documentation to support PA

Background

Background: Neuronal ceroid lipofuscinosis type 2 (CLN2) is a rare, inherited lysosomal disorder caused by deficiency of the tripeptidyl peptidase 1 (TPP1) enzyme. Clinically, affected children typically present in early childhood with progressive motor decline, language impairment, seizures, and cerebellar signs leading to loss of ambulation over time. Cerliponase alfa is an intraventricular TPP1 replacement therapy intended to slow loss of ambulation in symptomatic patients; policy review and coverage determinations for indications outside those specified here are guided by available evidence and expert review.

Definitions

TPP1 deficiency — laboratory demonstration of reduced enzyme activity in leukocytes or fibroblasts

Laboratory demonstrationDeficiency of tripeptidyl peptidase 1 (TPP1) in leukocytes or fibroblasts

Specimen typesLeukocytes or fibroblasts may be used to measure TPP1 activity

Role in diagnosisOne of two acceptable diagnostic confirmations for CLN2 required for prior authorization

Biallelic pathogenic/likely pathogenic TPP1 variants — genetic test result demonstrating two pathogenic/likely pathogenic variants

Genetic confirmationDemonstration of biallelic pathogenic or likely pathogenic variants in the TPP1 gene

InterpretationTwo pathogenic or likely pathogenic variants consistent with CLN2 disease satisfy diagnostic requirement