BlueCross BlueShield of Tennessee Medical Policy Manual

Agalsidase Beta

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 from resultant 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.  

An example of a preparation of agalsidase beta is FabrazymeŽ.

REFER TO DECISION SUPPORT TREE

POLICY

MEDICAL APPROPRIATENESS

APPLICABLE TENNESSEE STATE MANDATE REQUIREMENTS

Tennessee State law requires coverage of off-label indications of Food and Drug Administration (FDA) approved drugs when the off-label use is relative to life-threatening illnesses, such as cancer, AIDS, and coronary heart disease and recognized in one of the standard reference compendia (As defined in the statute:  The United States Pharmacopoeia Drug Information, The American Medical Association Drug Evaluations, & The American Hospital Formulary Service Drug Information) or in the medical literature. This law is applicable to all fully insured members. The law is not applicable to self-funded accounts, but coverage for off-label uses may be provided based on the contractual agreement.  

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 dosage information, contraindications, precautions, warnings, and monitoring information, please refer to one of the standard reference compendia (e.g., The American Hospital Formulary Service Drug Information).

There is insufficient evidence supporting the use of agalsidase beta for the treatment of other conditions/diseases.

SOURCES

Lexi-Comp Online. (2011). AHFS DI. Agalsidase beta. Retrieved February 15, 2011 from Lexi-Comp Online with AHFS.

MICROMEDEX Healthcare Series. Drugdex Drug Evaluations. (2010, July). Agalsidase beta. Retrieved February 15, 2011 from MICROMEDEX Healthcare Series.

U. S. Food and Drug Administration. Center for Drug Evaluation and Research. (2010, May). FabrazymeŽ (agalsidase beta). Retrieved May 25, 2009 from http://www.accessdata.fda.gov/drugsatfda_docs/label/2010/103979s5135lbl.pdf.

Wraith, J. E., Tylki-Szymanska, A., Guffon, N., Lien, Howard, Tismaratos, M., Vellodi, A., et al. (2007). Safety and efficacy of enzyme replacement therapy with agalsidase beta: An international, open-label study in pediatric patients with Fabry disease. The Journal of Pediatrics, 152 (4), 563-570. (Level of Evidence - III)

ORIGINAL EFFECTIVE DATE:  2/1/2005

MOST RECENT REVIEW DATE:  4/12/2011

ID_BT

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 (FabrazymeŽ)

  1. Does the individual have a diagnosis of Fabry disease?

      2. If yes, this satisfies medical necessity and medical appropriateness criteria

      If no, go to question #2

  2. Is the individual a carrier of Fabry disease and exhibits significant manifestations (e.g., 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, or acroparesthesias)?

      3. 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.