Alglucosidase Alfa
DESCRIPTION
Alglucosidase alfa is a glycoprotein which consists of the recombinant form of the human lysosomal enzyme acid α-glucosidase (GAA, acid maltase) that is encoded by the most predominant of nine observed haplotypes of the GAA genetic sequence. GAA is required for glycogen cleavage in the human body. The absence or marked deficiency of GAA results in Pompe disease (glycogen storage disease type II, GSD II, glycogenosis type II, acid maltase deficiency). It is an inherited progressive metabolic neuromuscular disorder of glycogen metabolism. Enzyme replacement therapy with Alglucosidase alfa (rhGAA) is used to treat Pompe disease.
Pompe disease is categorized as early onset (i.e., infantile Pompe disease) or late onset (i.e., juvenile/adult) Pompe disease by the National Institute of Neurological Disorders and Stroke (NINDS). Previous terminology defined three classifications: infantile-onset, juvenile-onset and adult-onset. Without enzyme replacement therapy the disease is terminal in infants within their first year, generally due to cardiorespiratory failure or respiratory infection. Prognosis for late onset disease depends upon the age at onset and is generally more serious in children and teens; however ultimately, prognosis is dependent upon the extent of respiratory muscle involved.
Examples of preparations of alglucosidase alfa are Myozyme® and Lumizyme™.
REFER TO DECISION SUPPORT TREE
POLICY
Alglucosidase alfa for the treatment of Pompe disease is considered medically necessary if the medical appropriateness criteria are met. (See Medical Appropriateness below.)
Alglucosidase alfa for the treatment of other conditions/diseases is considered investigational.
MEDICAL APPROPRIATENESS
Alglucosidase alfa for the treatment of Pompe disease is considered medically appropriate if ANY ONE of the following criteria are met:
Myozyme® is the agent being used for the treatment of early onset (infantile-onset) Pompe disease
Lumizyme™ is the agent being used for the treatment of late onset (i.e., juvenile/adult) Pompe disease with ALL the following:
The individuals is 8 years of age or older
Has no evidence of cardiac hypertrophy
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 alglucosidase alfa for the treatment of other conditions/diseases.
SOURCES
MICROMEDEX Healthcare Series. Drugdex Drug Evaluations. (2010). Alglucosidase alfa. Retrieved May 27, 2010 from MICROMEDEX Healthcare Series.
National Institute of Neurological Disorders and Stroke. (2010, May). NINDS Pompe Disease Information Page. Retrieved May 27, 2010 from http://www.ninds.nih.gov/disorders/pompe/pompe.htm.
U. S. Food and Drug Administration. (2010, May). Center for Drug Evaluation and Research. Lumizyme™ (alglucosidase alfa). Retrieved May 27, 2010 from http://www.accessdata.fda.gov/drugsatfda_docs/label/2010/125291lbl.pdf.
U. S. Food and Drug Administration. (2008, December). Center for Drug Evaluation and Research. Myozyme® (alglucosidase alfa). Retrieved November 17, 2009 from http://www.accessdata.fda.gov/drugsatfda_docs/label/2008/125141_74lbl.pdf.
ORIGINAL EFFECTIVE DATE: 1/13/2007
MOST RECENT REVIEW DATE: 8/3/2010
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
Alglucosidase alfa (Myozyme®, Lumizyme™)
Myozyme®
Is Myozyme® being requested for the treatment of early onset (i.e., infantile-onset) Pompe disease?
If yes, this satisfies medical necessity and medical appropriateness criteria
If no go to question #2
Lumizyme™
Is Lumizyme™ being requested for the treatment of late onset (i.e., juvenile/adult) Pompe disease with ALL the following?
The individuals is 8 years of age or older
Has no evidence of cardiac hypertrophy
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.