58468-0070-XX - Aldurazyme 2.9 MG/5ML SOLN (GENZYME)
Laronidase is a recombinant form of the human enzyme L-iduronidase (alpha-L-iduronidase). L-iduronidase is a lysosomal enzyme that is necessary for the degradation of glycosaminoglycans to its substrates dermatan sulfate and heparan sulfate. Without this enzyme these substrates accumulate throughout the body leading to widespread cellular, tissue and organ dysfunction.
Individuals with inherited deficiency of L-iduronidase have the lysosomal storage disease mucopolysaccharidosis type I. Treatment with laronidase reverses the metabolic and pathologic abnormalities outside the central nervous system. Mucopolysaccharidosis type I is classified into three distinct subgroups:
Hurler's syndrome -most severe form, with neurologic, skeletal, and visceral involvement, including hepatosplenomegaly, cardiac disease, airway obstruction, mental retardation/development delay, corneal clouding, and severe skeletal abnormalities; death often occurs before the age of ten.
Hurler-Scheie syndrome - intermediate form characterized by slower progression of same types of complications, but with minimal-to-no mental retardation; death is usually later (e.g., 20s).
Scheie's syndrome - least severe with less extensive disease; some individuals may have a normal life span.
Laronidase for the treatment of Mucopolysaccharidosis type I is considered medically necessary if the medical appropriateness criteria are met. (See Medical Appropriateness below.)
Laronidase for the treatment of other conditions/diseases is considered investigational.
Laronidase is considered medically appropriate if ALL of the following criteria are met:
Diagnosis of Mucopolysaccharidosis type I confirmed by ANY ONE of the following:
Detection of pathogenic mutations in the IDUA gene by molecular genetic testing
Detection of deficient activity of the lysosomal enzyme α-L-iduronidase (IDUA);
Disease is further classified as ANY ONE of the following forms of the disease:
Hurler's syndrome or Severe MPS I
Hurler-Scheie syndrome or Attenuated MPS I
Scheie's syndrome or Attenuated MPS I with moderate to severe symptoms
Documented baseline value for urinary glycosaminoglycan (uGAG)
Documented baseline values for ANY ONE of the following:
Six months to less than 6 years of age with urinary glycosaminoglycan excretion, cardiac status, upper airway obstruction during sleep, growth velocity, or mental development, forced vital capacity (FVC), and/or 6-minute walk test
6 years of age and older with percent predicted FVC, 6-minute walk test, joint range of motion, left ventricular hypertrophy, growth, quality of life (CHAQ/HAQ/MPS HAQ)
Laronidase is considered medically appropriate for renewal if ALL of the following criteria are met:
Absence of unacceptable toxicity from the agent, such as severe hypersensitivity reactions; acute respiratory complications; acute cardiorespiratory reactions; severe infusion reactions, etc.
Individual has a documented reduction in uGAG levels
Individual has demonstrated a beneficial response to therapy compared to pretreatment baseline in ANY ONE of the following:
Individual 6 months to less than 6 years old with urinary glycosaminoglycan excretion, stability or improvement in cardiac status, upper airway obstruction during sleep, growth velocity, mental development, FVC and/or 6-minute walk test
Individual 6 years old or greater with stability or improvement in percent predicted FVC and/or 6-minute walk test, increased joint range of motion, decreased left ventricular hypertrophy, improved growth, improved quality of life (clinically meaningful change in the CHAQ/HAQ/MPS HAQ disability index)
DOSAGE & ADMINISTRATION
Mucopolysaccharidosis I (MPS I)
0.58 mg/kg of body weight administered once weekly (by intravenous infusion) over 3-4 hours.
LENGTH OF AUTHORIZATION
Coverage will be provided for twelve months and may be renewed.
Refer to DOSAGE LIMITS below
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).
Clarke, L. A., (2002, October Updated 2016, February). Mucopolysaccharidosis Type I. In: Adam, M. P., Ardinger, H. H., Pagon, R. A., et al., eds., GeneReviews®. Seattle (WA): University of Washington, Seattle; 1993-2018. Retrieved April 5, 2018 from https://www.ncbi.nlm.nih.gov/books/NBK1162/.
Lexicomp Online. (2018, February). AHFS DI. Laronidase. Retrieved April 5, 2018 from Lexicomp Online with AHFS DI.
MICROMEDEX Healthcare Series. Drugdex Evaluations. (2018, October). Laronidase. Retrieved February 11, 2019 from MICROMEDEX Healthcare Series.
U. S. Food and Drug Administration. (2013, April). Center for Drug Evaluation and Research. Aldurazyme (laronidase). Retrieved February 11, 2019 from http://www.accessdata.fda.gov/drugsatfda_docs/label/2013/125058s220lbl.pdf.
ORIGINAL EFFECTIVE DATE: 2/1/2005
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.
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 = 0.1 mg