BlueCross BlueShield of Tennessee Medical Policy Manual

Laronidase (Aldurazyme®)

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 or government program (e.g., TennCare), the express terms of the health plan or government program will govern.

POLICY          

INDICATIONS

The indications below including FDA-approved indications and compendial uses are considered a covered benefit provided that all the approval criteria are met and the member has no exclusions to the prescribed therapy.

FDA-Approved Indications

Aldurazyme is indicated for the treatment of adult and pediatric patients with Hurler and Hurler-Scheie forms of Mucopolysaccharidosis I (MPS I) and for patients with the Scheie form who have moderate to severe symptoms.

Limitations of Use

• The safety and effectiveness of treating mildly affected patients with the Scheie form have not been established.
• The effect of Aldurazyme on central nervous system manifestations of the disorder has not been determined.

All other indications are considered experimental/investigational and not medically necessary.

DOCUMENTATION

Submission of the following information is necessary to initiate the prior authorization review:

• Initial requests: alpha-L-iduronidase enzyme assay or genetic testing results supporting diagnosis.
• Continuation requests: chart notes documenting a clinically positive response to therapy, which shall include improvement, stabilization, or slowing of disease progression.

PRESCRIBER SPECIALITIES

This medication must be prescribed by or in consultation with a physician who specializes in the treatment of metabolic disease and/or lysosomal storage disorders.

COVERAGE CRITERIA

Mucopolysaccharidosis I (MPS I)

Authorization of 12 months may be granted for treatment of MPS I when both of the following criteria are met:

• Diagnosis of MPS I was confirmed by enzyme assay demonstrating a deficiency of alpha-L-iduronidase enzyme activity or by genetic testing.
• Member has one of the following:  

• The Hurler form (i.e., severe MPS I).
• The Hurler-Scheie form (i.e., attenuated MPS I).
• The Scheie form (Scheie syndrome; i.e., attenuated MPS I) with moderate to severe symptoms (e.g., normal intelligence, less progressive physical problems, corneal clouding, joint stiffness, valvular heart disease).

CONTINUATION OF THERAPY  

Authorization of 12 months may be granted for continued treatment in members requesting reauthorization for an indication listed in the Coverage Criteria section who have a clinically positive response to therapy, which shall include improvement, stabilization, or slowing of disease progression.

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.

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).

REFERENCES

1. Aldurazyme [package insert]. Cambridge, MA: Genzyme Corporation; December 2023.
2. Wraith JE, Clarke LA, Beck M, et al. Enzyme replacement therapy for mucopolysaccharidosis I: a randomized, double-blinded, placebo-controlled, multinational study of recombinant human alpha-L-iduronidase (laronidase). J Pediatr. 2004;144:581-588. 
3. Muenzer J, Wraith JE, Clarke LA; International Consensus Panel on Management and Treatment of Mucopolysaccharidosis I. Mucopolysaccharidosis I: management and treatment guidelines. Pediatrics. 2009 Jan;123(1):19-29.
4. Clarke LA. Mucopolysaccharidosis Type I. 2002 Oct 31 [Updated 2024 Apr 11]. In: Adam MP, Everman DB, Mirzaa GM, et al., editors. GeneReviews® [Internet]. Seattle (WA): University of Washington, Seattle; 1993-2023.  Accessed Jan 10, 2025.

ORIGINAL EFFECTIVE DATE: 2/1/2005

MOST RECENT REVIEW DATE: 9/30/2025

ID_CHS

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.