BlueCross BlueShield of Tennessee Medical Policy Manual

Galsulfase

NDC CODE(S)

68135-0020-01 - Naglazyme 1 MG/ML SOLN (BIOMARIN PHARMACEUTICALS)

DESCRIPTION

Galsulfase is a recombinant purified human enzyme.  Within the body, the natural enzyme, N-acetylgalactosamine 4-sulfatase, is a lysosomal glycosaminoglycan (GAG)-specific enzyme required for the removal of the GAG substrate, dermatan sulfate, throughout the body.  Without the natural enzyme, the accumulation of dermatan sulfate leads to widespread cellular, tissue and organ dysfunction.

Individuals with an absence or marked shortage of the endogenous enzyme are diagnosed with the mucopolysaccharide storage disorder of mucopolysaccharidosis VI (MPS VI or Maroteaux-Lamy syndrome). Clinical symptoms of MPS VI include skeletal deformities, organ and soft tissue involvement.  Specific abnormalities may include short stature, abnormal bone formation, degenerative joint disease, hydrocephalus, impaired vision and hearing, sleep disorders, reduced endurance, coarse facial features, carpal tunnel syndrome, spastic quadriplegia, hepatomegaly, splenomegaly, cardiac valve dysfunction, respiratory dysfunction and shortened life span to between childhood and early adulthood.

REFER TO DECISION SUPPORT TREE

POLICY

MEDICAL APPROPRIATENESS

INITIAL APPROVAL

RENEWAL CRITERIA

INDICATION(S) DOSAGE & ADMINISTRATION
Mucopolysaccharidosis VI (Maroteaux-Lamy syndrome) 1mg/kg intravenously infused over no less than 4 hours once a week

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 naglazyme for the treatment or prevention of other conditions or diseases.

SOURCES

Lexi-Comp Online. (2016). AHFS DI. Galsulfase. Retrieved October 31, 2016 from Lexi-Comp Online with AHFS.

MICROMEDEX Healthcare Series. Drugdex Drug Evaluations. (2016, August). Galsulfase. Retrieved October 31, 2016 from MICROMEDEX Healthcare Series.

U. S. Food and Drug Administration. (2013, March). Center for Drug Evaluation and Research. Naglazyme® (galsulfase) injection for intravenous use. Retrieved October 31, 2016 from http://www.accessdata.fda.gov/drugsatfda_docs/label/2013/125117s111lbl.pdf.

ORIGINAL EFFECTIVE DATE:  10/14/2006

MOST RECENT REVIEW DATE:  2/27/2018

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

Galsulfase (Naglazyme®)

  1. Is this the initial request for this agent?

If yes, go to question #2

If no, go to question #4

  1. Does the individual have a diagnosis of mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy syndrome) with documentation of ANY ONE of the following?

If yes, go to question #3

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

  1. Is the request for 115 billable units or less every 7 days for dosage of 1mg/kg intravenously infused over no less than 4 hours once a week for an authorization period of six 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. Is there evidence of disease response to treatment as defined by improvement in 12-minute walk test (12-MWT) or 3-minute stair climb test?

If yes, go to question #5

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

  1. Is there absence of unacceptable toxicity from the agent, including, but not limited to, anaphylaxis and allergic reactions; immune mediated reactions; acute respiratory complications; acute cardiorespiratory reactions; infusion reactions?

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

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

 

This document has been classified as public information.