58468-0041-XX FABRAZYME 5MG Solution Reconstituted (GENZYME)
58468-0040-XX FABRAZYME 35MG Solution Reconstituted (GENZYME)
Agalsidase beta is a recombinant DNA origin form of human α-galactosidase A, a lipid degrading enzyme. It has the same amino acid sequence as the native enzyme. It is used to treat Fabry disease, deficiency of the enzyme α-galactosidase A.
Fabry disease is an X-linked recessive inborn error of glycosphingolipid metabolism with an estimated frequency of about 1 in 50,000 births. The result of this mutation is progressive accumulation of glycosphingolipids in cellular lysosomes of multiple body tissues. Clinical manifestations typically begin in childhood and may include abdominal or flank pain simulating appendicitis or renal colic, angiokeratomas, hypohidrosis, corneal and lenticular opacities, vascular disease of the kidney, heart, and brain, intolerance to heat, cold, and exercise, mild proteinuria, gastrointestinal problems, and acroparesthesias. Fabry crises, lasting from minutes to several days, consist of agonizing, burning pain in the hands, feet, and proximal parts of the extremities. Affected individuals have a lifespan of 30 to 50 years, typically resultant from renal failure, hypertrophic cardiomyopathy, myocardial infarction or cerebrovascular accidents. Female carriers may be asymptomatic or may exhibit severe manifestations similar to males with classic disease.
Enzyme replacement therapy for Fabry disease with agalsidase beta has been shown to provide an exogenous source of α-galactosidase A. It can reverse histologic abnormalities as well as improve some clinical manifestations of the disease.
Agalsidase beta for the treatment of Fabry disease is considered medically necessary if the medical appropriateness criteria are met (See Medical Appropriateness below.)
Agalsidase beta for the treatment of other conditions/diseases is considered investigational.
Agalsidase beta is considered medically appropriate if ALL of the following criteria are met:
Individual is 8 years of age or older
Diagnosis of Fabry disease (alpha-galactosidase A deficiency) is documented with biochemical/genetic confirmation by ANY ONE of the following:
Plasma α-galactosidase A (α-Gal A) activity in plasma, isolated leukocytes, and/or cultured cells (in males only)
Plasma or urinary globotriaosyl-ceramide(Gb3/GL-3) or globotriaosylsphingosine (lyso-Gb3)
Detection of pathogenic mutations in the GALA/GLA gene by molecular genetic testing
Baseline value for plasma GL-3 and/or GL-3 inclusions
Must not be used in combination with migalastat
Agalsidase beta is considered medically appropriate for renewal if ALL of the following criteria are met:
Individual continues to meet initial approval criteria
Absence of unacceptable toxicity from the drug, e.g., severe hypersensitivity reactions, severe infusion site reactions, compromised cardiac function, etc.
Disease response with treatment as defined by a reduction in GL-3 and/or GL-3 inclusions compared to pre-treatment baseline
|INDICATION(S)||DOSAGE & ADMINISTRATION|
The recommended dosage of Fabrazyme is 1 mg/kg body weight infused every two weeks as an intravenous (IV) infusion.Individuals should receive antipyretics prior to infusion. The initial IV infusion rate should be no more than 0.25 mg/min (15 mg/hr). The infusion rate may be slowed in the event of infusion reactions. After patient tolerance to the infusion is well established, the infusion rate may be increased in increments of 0.05 to 0.08 mg/min (increments of 3 to 5 mg/hr) with each subsequent infusion. For patients weighing < 30 kg, the maximum infusion rate should remain at 0.25 mg/min (15 mg/hr). For patients weighing ≥ 30 kg, the administration duration should not be less than 1.5 hours (based on individual patient tolerability).
LENGTH OF AUTHORIZATION
Coverage will be provided for 12 months and may be renewed
Refer to DOSAGE LIMITS below
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.
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, the express terms of the health plan will govern.
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).
Lexi-Comp Online. (2019). AHFS DI. Agalsidase beta. Retrieved September 4, 2019 from Lexi-Comp Online with AHFS.
Mehta, A., Hughes, E. A. (2002, August, updated 2017, January). Fabry Disease. In: Adam, M. P., Ardinger, H. H., Pagon, R.A., et al., eds., GeneReviews® [Internet]. Seattle (WA): University of Washington, Seattle; 1993-2018. Retrieved November 7, 2018 from https://www.ncbi.nlm.nih.gov/books/NBK1292/.
MICROMEDEX Healthcare Series. Drugdex Drug Evaluations. (2019, January). Agalsidase beta. Retrieved September 4, 2019 from MICROMEDEX Healthcare Series.
U. S. Food and Drug Administration. Center for Drug Evaluation and Research. (2018, December). Fabrazyme® (agalsidase beta). Retrieved September 4, 2019 from https://www.accessdata.fda.gov/drugsatfda_docs/label/2018/103979s5303lbl.pdf.
U. S. Food and Drug Administration. Center for Drug Evaluation and Research. (2019, August). Galafold™ (migalastat) capsules, for oral use. Retrieved September 4, 2019 from https://www.accessdata.fda.gov/drugsatfda_docs/label/2019/208623s001lbl.pdf.
ORIGINAL EFFECTIVE DATE: 2/1/2005
MOST RECENT REVIEW DATE: 10/8/2019
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.
Maximum billable units per dose and over time by indication as a Medical Benefit; 1 billable unit = 1 mg