IMPORTANT REMINDER
We develop Medical Policies to provide guidance to Members and Providers. This Medical Policy relates only to the services or supplies described in it. The existence of a Medical Policy is not an authorization, certification, explanation of benefits or a contract for the service (or supply) that is referenced in the Medical Policy. For a determination of the benefits that a Member is entitled to receive under his or her health plan, the Member's health plan must be reviewed. If there is a conflict between the Medical Policy and a health plan or government program (e.g., TennCare), the express terms of the health plan or government program will govern.
POLICY
I. INDICATIONS
The indications below including FDA-approved indications and compendial uses are considered a covered benefit provided that all the approval criteria are met and the member has no exclusions to the prescribed therapy.
FDA-Approved Indication
Brineura is indicated to slow the loss of ambulation in symptomatic pediatric patients 3 years of age and older with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2), also known as tripeptidyl peptidase 1 (TPP1) deficiency.
All other indications are considered experimental/investigational and not medically necessary.
II. DOCUMENTATION
Submission of the following information is necessary to initiate the prior authorization review: tripeptidyl peptidase 1 (TPP1) enzyme assay or genetic testing results supporting diagnosis.
III. CRITERIA FOR INITIAL APPROVAL
Late infantile neuronal ceroid lipofuscinosis type 2 (CLN2)
Authorization of 12 months may be granted for members with CLN2 when all of the following criteria are met:
1. Diagnosis of CLN2 was confirmed by enzyme assay demonstrating a deficiency of tripeptidyl peptidase 1 (TPP1) enzyme activity or by genetic testing; and
2. Member is 3 years of age or older; and
3. Brineura will be administered by, or under the direction of a physician knowledgeable in intraventricular administration; and
4. Dosage of Brineura will not exceed 300 mg once every other week; and
5. Member does not have acute intraventricular access device-related complications (e.g., leakage, device failure, or device-related infection) or a ventriculoperitoneal shunt.
IV. CONTINUATION OF THERAPY
Authorization of 12 months may be granted for continued treatment in members requesting reauthorization for an indication listed in Section III when the following criteria are met:
A. Member has experienced no loss of ambulation or a slowed loss of ambulation from baseline; and
B. Member does not have acute intraventricular access device-related complications (e.g., leakage, device failure, or device-related infection) or ventriculoperitoneal shunts.
APPLICABLE TENNESSEE STATE MANDATE REQUIREMENTS
BlueCross BlueShield of Tennessee’s Medical Policy complies with Tennessee Code Annotated Section 56-7-2352 regarding coverage of off-label indications of Food and Drug Administration (FDA) approved drugs when the off-label use is recognized in one of the statutorily recognized standard reference compendia or in the published peer-reviewed medical literature.
ADDITIONAL INFORMATION
For appropriate chemotherapy regimens, dosage information, contraindications, precautions, warnings, and monitoring information, please refer to one of the standard reference compendia (e.g., the NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®) published by the National Comprehensive Cancer Network®, Drugdex Evaluations of Micromedex Solutions at Truven Health, or The American Hospital Formulary Service Drug Information).
REFERENCES
1. Brineura [package insert]. Novato, CA: BioMarin Pharmaceutical, Inc.; March 2020.
2. Fietz M, AlSayed M, Burke, D, et al. Diagnosis of neuronal ceroid lipofuscinosis type 2 (CLN2 disease): Expert recommendations for early detection and laboratory diagnosis. Molecular Genetics and Metabolism. 2016 (11): 160-167.
ORIGINAL EFFECTIVE DATE: 7/28/2017
MOST RECENT REVIEW DATE: 1/1/2024
ID_CHS
Policies included in the Medical Policy Manual are not intended to certify coverage availability. They are medical determinations about a particular technology, service, drug, etc. While a policy or technology may be medically necessary, it could be excluded in a member's benefit plan. Please check with the appropriate claims department to determine if the service in question is a covered service under a particular benefit plan. Use of the Medical Policy Manual is not intended to replace independent medical judgment for treatment of individuals. The content on this Web site is not intended to be a substitute for professional medical advice in any way. Always seek the advice of your physician or other qualified health care provider if you have questions regarding a medical condition or treatment.
This document has been classified as public information.