CurrentNeighborhood Health Plan of Rhode IslandPolicy N/A

Brineura (cerliponase alfa) intraventricular therapy for CLN2 disease

Policy governs coverage, clinical criteria, dosing, administration, renewal, billing codes, and allowable units for Brineura (cerliponase alfa) for Medicaid, Commercial, and Medicare members of Neighborhood Health Plan of Rhode Island.

Policy Summary

PayerNeighborhood Health Plan of Rhode Island

PolicyBrineura (cerliponase alfa) intraventricular therapy for CLN2 disease

Policy CodePolicy N/A

Change TypeClarified dates (no material change)

Effective DateJan 1, 2020

Next Review DateMay 7, 2025

Key ActionObtain prior authorization by documenting definitive CLN2 diagnosis (TPP1 enzyme deficiency or two pathogenic TPP1/CLN2 variants), Hamburg CLN2 scores, device/infection status, and request authorization for 6 months; renew if renewal criteria met.

POLICY UPDATE CHANGES

Effective date and review dates provided; scope includes Medicaid, Commercial, Medicare.

6 monthsLength of authorization

300 / 14dMax units per dosing interval

E75.4Primary covered diagnosis (ICD-10)

J0567HCPCS J-code

Coverage Summary

Brineura (cerliponase alfa) is an intraventricular enzyme replacement therapy indicated to slow the loss of ambulation in pediatric patients with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2/TPP1 deficiency). Clinical trials demonstrated benefits in slowing decline in motor and language function and reducing seizure frequency. Common adverse reactions reported include pyrexia, ECG abnormalities, increased CSF protein, vomiting, seizures, hypersensitivity, headache, irritability, pleocytosis, device-related infection, bradycardia, and hypotension. The policy scope covers Medicaid, Commercial, and Medicare members.

Key policy stats and thresholds

Length of authorization6 months

Max units per dosing interval300 units / 14 days

Primary covered diagnosis (ICD-10)E75.4

HCPCS J-codeJ0567

Hamburg CLN2 score thresholdTwo-domain motor + language score 3–6 with ≥1 in each domain

Gestational / weight threshold>= 37 weeks post‑menstrual age; >= 2.5 kg

ECG recency requirement12‑lead ECG within last 6 months for renewal (or ECG during each infusion if cardiac abnormalities/history present)

Provider Actions & Requirements

Prior Authorization

Prior authorization required

Coverage requires meeting initial approval criteria. Authorization is provided for 6 months and may be renewed if renewal criteria are met.

Documentation Required

Diagnostic confirmation documentation

Provider must document a definitive diagnosis of CLN2: deficiency of the lysosomal enzyme TPP1 and/or molecular analysis showing two pathogenic variants/mutations in the TPP1/CLN2 gene, and document motor and language domain scores on the Hamburg CLN2 Clinical Rating Scale.

Documentation Required

Device and infection status documentation

Document absence of acute intraventricular access device-related complications, absence of ventriculoperitoneal (VP) shunt, and no signs or symptoms of acute, unresolved localized infection on or around the device insertion site or suspected/confirmed CNS infection prior to infusion.

Billing Rule

Unit limits and dosing frequency

Maximum 300 billable units (one kit containing 2 vials) every 14 days. J0567 is billed per 1 mg unit consistent with dosing.

Documentation Required

ECG monitoring requirement

ECG monitoring must be performed during each infusion for patients with a history of bradycardia, conduction disorder, or structural heart disease. For renewal, a 12-lead ECG must have been performed within the last 6 months (patients with cardiac abnormalities require ECG each infusion).

Billing Rule

Administration setting and device implantation timing

Brineura must be administered via a surgically implanted intraventricular access device. The first dose is recommended at least 5 to 7 days after device implantation. Administration should be supervised in a healthcare setting equipped to manage anaphylaxis and provide cardiopulmonary resuscitation/resuscitation support.

Clinical Evidence & References

Key evidence includes the randomized/clinical study by Schulz et al. (Study of Intraventricular Cerliponase Alfa for CLN2 Disease; N Engl J Med. 2018) and the Brineura prescribing information (BioMarin package insert, August 2024).

Trial-level outcomes reported in the evidence include slowing of motor and language decline and reduction in seizure frequency in patients with CLN2 disease. The package insert and policy summary also document common adverse reactions and safety findings associated with intraventricular administration of cerliponase alfa.

Background & Definitions

Brineura (cerliponase alfa) is administered by intraventricular infusion via a surgically implanted intraventricular access device and is an enzyme replacement for TPP1 deficiency in pediatric CLN2 disease. It should be given in a monitored healthcare setting by clinicians experienced in intraventricular administration, with precautions for hypersensitivity and anaphylaxis. Adverse reactions reported include pyrexia, ECG abnormalities, increased CSF protein, vomiting, seizures, hypersensitivity, device-related infection, and bradycardia.

Term	Definition
Universal Criteria	Patient is at least 37 weeks post‑menstrual age and weighs at least 2.5 kg; must not have any acute intraventricular access device‑related complications (e.g., extravasation of fluid, device failure, leakage); must not have ventriculoperitoneal shunts; and must have no signs or symptoms of acute, unresolved localized infection on or around the device insertion site (e.g., cellulitis or abscess) or a suspected or confirmed CNS infection (e.g., cloudy CSF, positive CSF gram stain, or meningitis)
Hamburg CLN2 Clinical Rating Scale	Scale used to assess motor and language domains; eligibility requires a two‑domain score of 3 to 6 on the motor and language domains with a score of at least 1 in each domain (used to document mild to moderate symptoms)

Term

Definition

Universal Criteria

Patient is at least 37 weeks post‑menstrual age and weighs at least 2.5 kg; must not have any acute intraventricular access device‑related complications (e.g., extravasation of fluid, device failure, leakage); must not have ventriculoperitoneal shunts; and must have no signs or symptoms of acute, unresolved localized infection on or around the device insertion site (e.g., cellulitis or abscess) or a suspected or confirmed CNS infection (e.g., cloudy CSF, positive CSF gram stain, or meningitis)

Hamburg CLN2 Clinical Rating Scale

Scale used to assess motor and language domains; eligibility requires a two‑domain score of 3 to 6 on the motor and language domains with a score of at least 1 in each domain (used to document mild to moderate symptoms)

Revision History

2020-01-01effective

Effective Date documented as 01/01/2020.

2024-08-28last_review

Last review date documented as 08/28/2024.

2025-05-07next_reviewLatest

Next review date documented as 05/07/2025.

2020-01-01clarified

Document shows Effective Date 01/01/2020 and review dates 08/28/2024 and 05/07/2025, but policy statements unchanged.

Policy Summary

PayerNeighborhood Health Plan of Rhode Island

PolicyBrineura (cerliponase alfa) intraventricular therapy for CLN2 disease

Policy CodePolicy N/A

Change TypeClarified dates (no material change)

Effective DateJan 1, 2020

Next Review DateMay 7, 2025

On This Page

OpenPayer

Brineura (cerliponase alfa) intraventricular therapy for CLN2 disease

Coverage Summary

Initial Approval Criteria

Renewal / Continuation Criteria

Provider Actions & Requirements

Applicable Codes

Clinical Evidence & References

Background & Definitions

Revision History