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
Kanuma is indicated for the treatment of patients with a diagnosis of Lysosomal Acid Lipase (LAL) 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:
A. Initial requests: lysosomal acid lipase enzyme assay or genetic testing results supporting diagnosis.
B. Continuation requests: lab values or chart notes documenting a positive response to therapy (e.g., improvement, stabilization, or slowing of disease progression for weight-for-age z-score if exhibiting growth failure, LDL, HDL, triglycerides, or ALT).
III. CRITERIA FOR INITIAL APPROVAL
Lysosomal acid lipase (LAL) deficiency
Authorization of 12 months may be granted for treatment of LAL deficiency when both of the following criteria are met:
A. Diagnosis of LAL deficiency was confirmed by enzyme assay demonstrating a deficiency of lysosomal acid lipase enzyme activity or by genetic testing; AND
B. Member has alanine aminotransferase level (ALT) ≥ 1.5 times the upper limit of normal (based on the age- and gender-specific normal ranges) on two consecutive ALT measurements obtained at least one week apart.
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 who are responding to therapy (e.g., improvement, stabilization, or slowing of disease progression for weight-for-age z-score if exhibiting growth failure, low-density lipoprotein [LDL], high-density lipoprotein [HDL], triglycerides, or alanine aminotransferase [ALT]).
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. Kanuma [package insert]. Boston, MA: Alexion Pharmaceuticals Inc.; November, 2021.
2. Burton BK, Balwani, M, Feillet F, et al. A Phase 3 Trial of Sebelipase Alfa in Lysosomal Acid Lipase Deficiency. N Engl J Med.2015;373:1010-20.
ORIGINAL EFFECTIVE DATE: 1/6/2016
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.