58468-0160-XX Lumizyme 50 mg injection (Genzyme Corporation)
Alglucosidase alfa is an enzyme-replacement agent produced by recombinant DNA technology. It consists of the human enzyme acid α-glucosidase (GAA) that is encoded by the nine most predominant observed haplotypes of this genetic sequence. GAA is a glycogen-specific enzyme necessary for the degradation or cleavage of glycogen within lysosomes, organelles within cells which degrade proteins, membranes and ingested materials. Individuals unable to produce GAA or who produce insufficient GAA have an inherited disorder of glycogen metabolism known as Pompe disease. It is also referred to as acid maltase deficiency, glycogen storage disease type II, GSD II or glycogenosis type II. This autosomal recessive disorder results in the accumulation of lysosomal glycogen in multiple tissues and cell types, most seriously affecting cardiac, skeletal and smooth muscle cells.
Pompe disease is generally considered in two forms. Early onset or infantile-onset Pompe disease is fatal for infants who do not receive enzyme replacement therapy. Symptoms of hypotonia and generalized muscle weakness are noted in the first months of life with rapid progression to death due to cardiorespiratory failure or respiratory infection within their first year. Late onset or juvenile/adult Pompe disease does not typically have cardiac involvement but results in skeletal muscle dysfunction progressive to inability to walk, ventilator support and eventual death from respiratory failure without enzyme replacement therapy.
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 or prevention of other conditions/diseases is considered investigational.
Alglucosidase alfa is considered medically appropriate if ALL of the following criteria are met:
Diagnosis of Pompe Disease (Acid alpha-glucosidase or GAA deficiency) if ALL of the following:
Confirmed by ANY ONE of the following:
Deficiency of acid alpha-glucosidase enzyme activity
Detection of pathogenic variants in the GAA gene by molecular genetic testing
Documented baseline values for ANY ONE of the following:
Infantile-onset disease with documentation of muscle weakness, motor function, respiratory function, cardiac involvement, percent predicted forced vital capacity (FVC), and/or 6 minute walk test (6MWT)
Late-onset (non-infantile) with documentation of percent predicted forced vital capacity (FVC), and/or 6 minute walk test (6MWT)
Alglucosidase alfa is considered medically appropriate for renewal if ALL of the following criteria are met:
Individual continues to meet initial approval criteria
Absence of unacceptable toxicity from the drug, e.g., severe allergic and anaphylactic reactions, severe cutaneous and systemic immune mediated reactions, acute cardiorespiratory failure, cardiac arrhythmia and sudden cardiac death during general anesthesia, etc.
No evidence that patient has developed IgG antibodies to alglucosidase alfa at a sustained titer level of > 12,800
Individual has demonstrated a beneficial response to therapy compared to pretreatment baseline in one or more of the following:
Infantile-onset disease: (e.g. decrease stabilization or improvement in muscle weakness, motor function, improvement in respiratory function, decreased cardiac involvement, FVC, and/or 6MWT
Late-onset (non-infantile) disease: stabilization or improvement in FVC and/or 6MWT
|INDICATION(S)||DOSAGE & ADMINISTRATION|
20mg/kg body weight as an intravenous (IV) infusion every 2 weeks
LENGTH OF AUTHORIZATION
Coverage will be provided for 12 months and may be renewed
Refer to DOSAGE LIMITS below
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.
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.
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).
Leslie, N., Bailey, L. (2007, August updated 2017, May). Pompe Disease. Adam, M. P., Ardinger, H. H., Pagon, R. A., et al., eds., GeneReviews® (Internet). Seattle (WA): University of Washington, Seattle; 1993-2018. Retrieved April 17, 2018 from https://www.ncbi.nlm.nih.gov/books/NBK1261/.
Lexi-Comp Online. (2019, February). AHFS DI. Alglucosidase alfa. Retrieved March 1, 2019 from Lexi-Comp Online with AHFS.
MICROMEDEX Healthcare Series. Drugdex Drug Evaluations. (2018, October). Alglucosidase alfa. March 1, 2019 from MICROMEDEX Healthcare Series.
U. S. Food and Drug Administration. (2014, August). Center for Drug Evaluation and Research. Lumizyme® (alglucosidase alfa). Retrieved March 1 2019 from http://www.accessdata.fda.gov/drugsatfda_docs/label/2014/125291s136lbl.pdf.
ORIGINAL EFFECTIVE DATE: 1/13/2007
MOST RECENT REVIEW DATE: 4/9/2019
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.
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Maximum billable units per dose and over time by indication as a Medical Benefit; 1 billable unit = 10 mg