BlueCross BlueShield of Tennessee Medical Policy Manual

Alglucosidase Alfa

NDC CODE(S)

Lumizyme 50 mg injection - 58468-0160-xx (Genzyme Corporation)

DESCRIPTION

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.

REFER TO DECISION SUPPORT TREE

POLICY

MEDICAL APPROPRIATENESS

INITIAL APPROVAL

RENEWAL CRITERIA

INDICATION(S) DOSAGE & ADMINISTRATION
Pompe disease

20mg/kg as an intravenous (IV) infusion every 2 weeks

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.

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 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).

No controlled studies were found in the published literature that validate the use of alglucosidase alfa for the prevention or treatment of other conditions or diseases.

SOURCES

Lexi-Comp Online. (2016). AHFS DI. Alglucosidase alfa. Retrieved September 14, 2016 from Lexi-Comp Online with AHFS.

MICROMEDEX Healthcare Series. Drugdex Drug Evaluations. (2016, August). Alglucosidase alfa. September 14, 2016  from MICROMEDEX Healthcare Series.

U. S. Food and Drug Administration. (2014, August). Center for Drug Evaluation and Research. Lumizyme® (alglucosidase alfa). Retrieved September 14, 2016 from http://www.accessdata.fda.gov/drugsatfda_docs/label/2014/125291s136lbl.pdf.

ORIGINAL EFFECTIVE DATE:  1/13/2007

MOST RECENT REVIEW DATE:  12/1/2016

ID_MRx

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 (Lumizyme®)

  1. Is this the initial request for this agent?

If yes, go to question #2

If no, go to question #5

  1. Does the individual have a diagnosis of Pompe disease (acid alpha-glucosidase deficiency)?

If yes, go to question #4

If no, go to question #3

  1. Does the individual have a diagnosis of Late-onset (non-infantile) with requested agent Lumizyme® and documentation of ALL of the following?

If yes, go to question #4

If no, this does not meet medical necessity and/or medical appropriateness criteria

  1. Is the request for 250 billable units or less every 15 days with a dosage of 20mg/kg as an intravenous (IV) infusion every 2 weeks for a period of 6 months?

If yes, this satisfies medical necessity and medical appropriateness criteria

If no, this does not meet medical necessity and/or medical appropriateness criteria

  1. Does the individual show ALL of the following?

If yes, this satisfies medical necessity and medical appropriateness criteria for 6 month renewal

If no, this does not meet medical necessity and/or medical appropriateness criteria

This document has been classified as public information.