Cerliponase Alfa (Brineura)

Policy ID: I-172-008

Section: Injections

Effective Date: July 01, 2018

Revised Date: January 11, 2024

Revision Effective Date: March 01, 2024

Last Reviewed: January 16, 2024

Applies To: Commercial Only

Description

Cerliponase alfa (Brineura™) is a hydrolytic lysosomal N-terminal tripeptidyl peptidase indicated to slow the loss of ambulation in symptomatic individuals three years of age and older with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2), also known as tripeptidyl peptidase 1 (TPP1) deficiency. CLN2 disease is one of a group of disorders known as neuronal ceroid lipofuscinoses, collectively called Battens disease. Deficiency in TPP1 activity results in the accumulation of lysosomal storage materials normally metabolized by this enzyme in the CNS, leading to progressive decline in motor function. Cerliponase alfa (Brineura), a proenzyme, is activated in the lysosome and cleaves tripeptides from the N-terminus of proteins.

Criteria

Coverage is subject to the specific terms of the member's benefit plan.

Federal Employee Program members (FEP) should check with their Retail Pharmacy Program to determine if prior approval is required by calling the Retail Pharmacy Program at 1-800-624-5060 (TTY: 1-800-624-5077). FEP members can also obtain the list through the www.fepblue.org website.

Cerliponase alfa (Brineura) may be considered medically necessary to slow the loss of ambulation in individuals with late infantile CLN2 who meet ALL of the following:

Individual is three (3) years of age or older; and
Individual is symptomatic (e.g., motor or language symptoms); and
Has a confirmed diagnosis of CLN2, defined as:
- Deficient TPP1 activity in leukocytes; and
- Pathogenic variants/mutations in each allele of TPP1/CLN2 gene; and
Cerliponase alfa (Brineura) is prescribed by or in consultation with a neurologist with experience and expertise in the treatment and ongoing management of individuals with CLN2; and
Documentation of current rating on the CLN2 Clinical Rating Scale.

Reauthorization Criteria

Continuation of treatment with cerliponase alfa (Brineura) after initiation of therapy, may be considered medically necessary to slow the loss of ambulation in individuals late infantile CLN2 who meet the following:

Documentation that individual has slowed the loss of ambulation from initial baseline CLN2 Clinical Rating Scale.

The use of cerliponase alfa (Brineura) for all other indications not listed in this policy is considered experimental/investigational and therefore non-covered because the safety and/or effectiveness cannot be established by the available published peer-reviewed literature.

Procedure Codes

J0567

NOTE: In addition to the above criteria, product specific dosage and/or frequency limits may apply in accordance with the U.S. Food and Drug Administration (FDA)-approved product prescribing information, national compendia, Centers for Medicare and Medicaid Services (CMS) and other peer reviewed resources or evidence-based guidelines.

Diagnosis Codes

E75.4

Professional Statements and Societal Positions Guidelines

Not Applicable

ND Committee Review

Internal Medical Policy Committee 1-14-2019 New code, J0567 added effective

Internal Medical Policy Committee 7-16-2019

Removed intent of treatment is to slow the loss of ambulation from initial criteria,
Revised continuation of therapy criteria

Internal Medical Policy Committee 7-22-2020 Annual review, no clinical content change

Internal Medical Policy Committee 11-19-2020 No clinical content change

Internal Medical Policy Committee 11-23-2021 Annual review, no clinical content change

Internal Medical Policy Committee 11-29-2022 Annual review, no clinical content change

Internal Medical Policy Committee 1-26-2023 Effective March 01, 2023

Updated experimental/investigational statement

Internal Medical Policy Committee 1-16-2024 Effective March 01, 2024

Annual review no clinical content change

Links

References (PDF)

Disclaimer

Current medical policy is to be used in determining a Member's contract benefits on the date that services are rendered. Contract language, including definitions and specific inclusions/exclusions, as well as state and federal law, must be considered in determining eligibility for coverage. Members must consult their applicable benefit plans or contact a Member Services representative for specific coverage information. Likewise, medical policy, which addresses the issue(s) in any specific case, should be considered before utilizing medical opinion in adjudication. Medical technology is constantly evolving, and the Company reserves the right to review and update medical policy periodically.

Policy ID: I-172-007