Pegunigalsidase Alfa-iwxj (Elfabrio®)
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
Elfabrio is indicated for the treatment of adults with confirmed Fabry disease.
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: alpha-galactosidase enzyme assay or genetic testing results supporting diagnosis. In the case of obligate carriers, the documentation must be submitted for the parent.
B. Continuation requests: lab results or chart notes documenting a positive response to therapy (e.g., reduction in plasma globotriaosylceramide [GL-3, Gb3] or GL-3/Gb3 inclusions, improvement and/or stabilization in renal function, pain reduction).
III. CRITERIA FOR INITIAL APPROVAL
Fabry disease
Authorization of 12 months may be granted for treatment of Fabry disease when both of the following criteria are met:
A. The diagnosis of Fabry disease was confirmed by enzyme assay demonstrating a deficiency of alpha-galactosidase enzyme activity or by genetic testing, or the member is a symptomatic obligate carrier; and
B. The requested medication will not be used in combination with Galafold.
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., reduction in plasma globotriaosylceramide [GL-3, Gb3] or GL-3/Gb3 inclusions, improvement and/or stabilization in renal function, pain reduction).
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. Elfabrio [package insert]. Cary, NC: Chiesi USA, Inc.; May 2023.
2. Biegstraaten M, Arngrimsson R, Barbey F, et al. Recommendations for initiation and cessation of enzyme replacement therapy in patients with Fabry disease: the European Fabry Working Group consensus document. Orphanet J Rare Dis. 2015; 1036.
3. Ortiz A, Germain DP, Desnick RJ, et al. Fabry disease revisited: Management and treatment recommendations for adult patients. Mol Genet Metab. 2018;123(4):416-427.
MOST RECENT REVIEW DATE: 9/10/2024
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.
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