Galsulfase (Systemic)
DESCRIPTION
Galsulfase (systemic) is a normal variant form of the polymorphic human enzyme N-acetylgalactosamine 4-sulfatase produced by recombinant DNA technology. This enzyme is a lysosomal hydrolase that catalyses the cleavage of the sulfate ester from terminal N-acetylgalactosamine 4-sulfate residues of glycosaminoglycans (GAG) chondroitin 4-sulfate and dermatan sulfate. Mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy syndrome) is the reduction or absence of N-acetylgalactosamine 4-sulfatase and results in accumulation of dermatan sulfate throughout the body, leading to widespread and progressive cellular, tissue, and organ dysfunction. Clinical manifestations include short stature, kyphosis, coarse facial features, dysostosis multiplex, joint stiffness, heart valve thickening, upper airway obstruction, hepatosplenomegaly, and corneal clouding. Lifespan of most patients is reduced to between childhood and early adulthood.
Galsulfase provides an exogenous enzyme that is taken up into lysosomes, increasing the catabolism of GAG. It has been shown to improve walking and stair-climbing capacity of patients with MPS VI.
An example of a preparation of galsulfase is Naglazyme®.
REFER TO DECISION SUPPORT TREE
POLICY
Galsulfase for the treatment of Mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy syndrome) is considered medically necessary.
Galsulfase for the treatment of other conditions/diseases is considered investigational.
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 galsulfase for the treatment of other conditions/diseases.
SOURCES
Lexi-Comp Online. (2009). AHFS DI. Galsulfase. Retrieved June 17, 2009 from Lexi-Comp Online with AHFS.
MICROMEDEX Healthcare Series. Drugdex Drug Evaluations. (2009). Galsulfase. Retrieved June 17, 2009 from MICROMEDEX Healthcare Series.
U. S. Food and Drug Administration. Center for Drug Evaluation and Research. (2005, June). FDA Labeling Information. Naglazyme™ (galsulfase). Retrieved June 17, 2009 from http://www.fda.gov/cder/foi/label/2005/125117_0000_lbl.pdf.
ORIGINAL EFFECTIVE DATE: 10/14/2006
MOST RECENT REVIEW DATE: 9/23/2009
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
Galsulfase (Naglazyme®)
Does the individual have a diagnosis of Mucopolysaccharidosis VI (MPS VI or Maroteaux-Lamy syndrome)?
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.