68135-0020-XX7 Naglazyme 1 MG/ML SOLN (BIOMARIN PHARMACEUTICALS)
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.
Galsulfase for the treatment of mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy syndrome) is considered medically necessary if the medical appropriateness criteria are met. (See Medical Appropriateness below.)
Galsulfase for the treatment of other conditions/diseases is considered investigational.
Galsulfase is considered medically appropriate if ALL of the following criteria are met:
Diagnosis of mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy syndrome) confirmed by ANY ONE of the following:
Detection of pathogenic mutations in the ARSB gene by molecular genetic testing
Arylsulfatase B (ASB) enzyme activity of <10% of the lower limit of normal in cultured fibroblasts or isolated leukocytes and ALL of the following:
Individual has normal enzyme activity of a different sulfatase (excluding patients with Multiple Sulfatase Deficiency [MSD])
Individual has an elevated urinary GAG level as defined as being above the upper limit of normal by the reference laboratory
Individual is 5 years of age or older
Documented baseline 12-minute walk test (12-MWT), 3-minute stair climb test, and/or pulmonary function tests (e.g., FEV1, etc.)
Documented baseline value for urinary glycosaminoglycan (uGAG)
Galsulfase is considered medically appropriate for renewal if ALL of the following criteria are met:
Individual continues to meet the initial approval criteria
Disease response to treatment as defined by reduction in uGAG levels and improvement or stability from pre-treatment baseline for ANY ONE of the following:
12-minute walk test (12-MWT)
3-minute stair climb test compared to pre-treatment baseline
Pulmonary function testing (e.g., FEV1, etc.)
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;severe infusion reactions, spinal or cervical cord compression, etc.
|INDICATION(S)||DOSAGE & ADMINISTRATION|
|Mucopolysaccharidosis VI (Maroteaux-Lamy syndrome)||
1mg/kg of body weight
administered as an intravenous
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).
Lexi-Comp Online. (2018). AHFS DI. Galsulfase. Retrieved November 28, 2018 from Lexi-Comp Online with AHFS.
MICROMEDEX Healthcare Series. Drugdex Drug Evaluations. (2017, August). Galsulfase. Retrieved November 28, 2018 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 March 28, 2018 from http://www.accessdata.fda.gov/drugsatfda_docs/label/2013/125117s111lbl.pdf.
Vairo F, Federhen A, Baldo G, et al. Diagnostic and treatment strategies in mucopolysaccharidosis VI. Appl Clin Genet. 2015 Oct 30;8:245-55.
ORIGINAL EFFECTIVE DATE: 10/14/2006
MOST RECENT REVIEW DATE: 4/2/2019
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.
This document has been classified as public information.
Maximum billable units per dose and over time by indication as a Medical Benefit 1 billable unit = 1 mg