Laronidase
DESCRIPTION
Laronidase is a recombinant form of the human enzyme L-iduronidase (alpha-L-iduronidase). L-iduronidase is a lysosomal enzyme that is necessary for the degradation of glycosaminoglycans to its substrates dermatan sulfate and heparan sulfate. Without this enzyme these substrates accumulate throughout the body leading to widespread cellular, tissue and organ dysfunction.
Individuals with inherited deficiency of L-iduronidase have the lysosomal storage disease Mucopolysaccharidosis type I. Treatment with laronidase reverses the metabolic and pathologic abnormalities outside the central nervous system. Mucopolysaccharidosis type I is classified into three distinct subgroups:
Hurler's syndrome - most severe form, with neurologic, skeletal, and visceral involvement, including hepatosplenomegaly, cardiac disease, airway obstruction, mental retardation/development delay, corneal clouding, and severe skeletal abnormalities; death often occurs before the age of ten.
Hurler-Scheie syndrome - intermediate form characterized by slower progression of same types of complications, but with minimal-to-no mental retardation; death is usually later (e.g., 20s).
Scheie's syndrome - least severe with less extensive disease; some individuals may have a normal life span.
An example of a preparation of laronidase is AldurazymeŽ.
REFER TO DECISION SUPPORT TREE
POLICY
Laronidase for the treatment of mucopolysaccharidosis type I is considered medically necessary if the medical appropriateness criteria are met. (See Medical Appropriateness below.)
Laronidase for the treatment of other conditions/diseases is considered investigational.
MEDICAL APPROPRIATENESS
Laronidase for the treatment of mucopolysaccharidosis type I is considered medically appropriate for ANY ONE of the following:
Hurler's syndrome
Hurler-Scheie syndrome
Scheie's syndrome with moderate to severe symptoms
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 in the printed literature to support of the use of laronidase for the treatment/prevention of other conditions/diseases.
SOURCES
Aldurazyme.com. (2010, May). Aldurazyme Prescribing Information. Retrieved February 2, 2011 from http://www.aldurazyme.com/pdf/az_us_hc_pi.pdf.
Lexi-Comp Online. (2011). AHFS DI. Laronidase. Retrieved February 2, 2011 from Lexi-Comp Online with AHFS.
MICROMEDEX Healthcare Series. Drugdex Evaluations. (2011). Laronidase. Retrieved February 2, 2011 from MICROMEDEX Healthcare Series.
U. S. Food and Drug Administration. (2010, May). Center for Drug Evaluation and Research. Alduradase (laronidase).Retrieved February 2, 2011 from http://www.accessdata.fda.gov/drugsatfda_docs/label/2010/125058s0186lbl.pdf.
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
Laronidase (AldurazymeŽ)
Does the individual have a diagnosis of mucopolysaccharidosis type I?
If yes, go to question #2
If no, this does not meet medical necessity and/or medical appropriateness criteria
Does the individual have a further diagnosis of ANY ONE of the following?
Hurler's syndrome
Hurler-Scheie syndrome
Scheie's syndrome with moderate to severe symptoms
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.