BlueCross BlueShield of Tennessee Medical Policy Manual

Agalsidase Beta

NDC CODE(S)

Fabrazyme 5 mg injection: 54868-0041-xx (Genzyme Corporation)

 

Fabrazyme 35 mg injection: 54868-0040-xx (Genzyme Corporation)

DESCRIPTION

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.

REFER TO DECISION SUPPORT TREE

POLICY

MEDICAL APPROPRIATENESS

INITIAL APPROVAL

RENEWAL CRITERIA

INDICATION(S) DOSAGE & ADMINISTRATION
Fabry Disease

·       The recommended dosage of Fabrazyme is 1 mg/kg body weight infused every two weeks as an intravenous (IV) infusion.

·       Patients 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).

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.

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, the express terms of the health plan will govern.

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).

No controlled studies were found in the published literature that validate the use of agalsidase beta for the treatment of other conditions or diseases.

SOURCES

Lexi-Comp Online. (2016). AHFS DI. Agalsidase beta. Retrieved September 14, 2016 from Lexi-Comp Online with AHFS.

MICROMEDEX Healthcare Series. Drugdex Drug Evaluations. (2016, August). Agalsidase beta. Retrieved September 14, 2016 from MICROMEDEX Healthcare Series.

U. S. Food and Drug Administration. Center for Drug Evaluation and Research. (2010, May). Fabrazyme® (agalsidase beta). Retrieved September 14, 2016 from http://www.accessdata.fda.gov/drugsatfda_docs/label/2010/103979s5135lbl.pdf.

ORIGINAL EFFECTIVE DATE:  2/1/2005

MOST RECENT REVIEW DATE:  12/1/2016

ID_MRx

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.

Pharmaceutical Decision Support Tree

Agalsidase Beta (Fabrazyme ®)

  1. Is this the initial request for the agent?

If yes, go to question #2

If no, go to question #5

  1. Is the individual 8 years of age or older?

If yes, go to question #3

If no, this does not meet medical necessity and/or medical appropriateness criteria

  1. Does the individual have a diagnosis of Fabry disease is documented with biochemical/genetic confirmation by ANY ONE of the following?

If yes, go to question #4

If no, this does not meet medical necessity and/or medical appropriateness criteria

  1. Is the request for 115 billable units or less every 14 days for dosage of 1mg/kg body weight infused every two weeks as an intravenous (IV) infusion for an authorization period of 12 months?

If yes, this satisfies medical necessity and medical appropriateness criteria

If no, this does not meet medical necessity and/or medical appropriateness criteria

  1. Does the individual show disease response to treatment as defined by a reduction in plasma glycosphingolipid GL-3 levels compared to baseline and an absence of unacceptable toxicity from the agent (e.g., hypersensitivity reactions) with the renewal as per question #4?

If yes, this satisfies medical necessity and medical appropriateness criteria

If no, this does not meet medical necessity and/or medical appropriateness criteria

This document has been classified as public information.