Anthem Blue Cross California Aldurazyme (laronidase) Form

Overview

This document addresses Aldurazyme (laronidase), an enzyme replacement therapy approved by the Food and Drug Administration (FDA) to treat individuals with Mucopolysaccharidosis I. Mucopolysaccharidosis I has historically been divided into three groups based on severity of symptoms: Hurler syndrome, Hurler-Scheie syndrome and Scheie syndrome. Aldurazyme is approved to treat all three syndromes with the exception of individuals exhibiting only mild symptoms of Scheie syndrome.

The mucopolysaccharidoses are a group of inherited metabolic diseases caused by the deficiency of lysosomal enzymes needed to breakdown mucopolysaccharides or glycosaminoglycan (GAGs). The progressive accumulation of GAGs in lysosomes leads to respiratory, cardiac, skeletal and connectivity, neurologic and ophthalmologic complications. There are seven distinct types of mucopolysaccharidosis (I, II, III, IV, VI, VII and IX). Accurate diagnosis is important to provide disease-specific enzyme replacement therapy. Diagnosis is confirmed through urinary GAG concentration measurement, enzymatic activity measurement or genetic testing.

Aldurazyme has a black box warning for anaphylaxis. Life-threatening anaphylactic reactions have occurred during Aldurazyme infusions. Appropriate medical support should be available during Aldurazyme administration. Individuals with compromised respiratory function or acute respiratory disease may be at risk of serious acute exacerbation of their respiratory disease and require additional monitoring.

Clinical Criteria

When a drug is being reviewed for coverage under a member's medical benefit plan or is otherwise subject to clinical review (including prior authorization), the following criteria will be used to determine whether the drug meets any applicable medical necessity requirements for the intended/prescribed purpose.

Aldurazyme (laronidase)

Initial requests for Aldurazyme (laronidase) may be approved if the following criteria are met:

1. Individual has a diagnosis of any of the following Mucopolysaccharidosis I (MPS I) syndromes:
   • Hurler syndrome; OR
   • Hurler-Scheie syndrome; OR
   • Documentation is provided for Scheie syndrome, moderate to severe symptoms including any of the following (Thomas 2010, Wang 2011):
     1. Cardiac valve abnormalities (including aortic or mitral valve regurgitation, with or without insufficiency or stenosis); OR
     2. Corneal clouding, open-angle glaucoma, and retinal degeneration, progressive; OR
     3. Craniofacial or growth retardation; OR
     4. Frequent, moderate to severe upper respiratory infections; OR
     5. Hepatosplenomegaly; OR
     6. Hernias (such as hiatal, inguinal, or umbilical); OR
     7. Neurological symptoms resulting from cervical instability or cervical spinal cord compression; OR
     8. Skeletal and joint involvement, progressive (including arthropathy, back pain, joint stiffness, lumbar spondylolisthesis, lumbar spinal compression, osteopenia, or osteoporosis); AND
2. Documentation is provided that diagnosis is demonstrated by one of the following (Clarke 2021, Lehman 2011):
   • Documented deficiency in alpha-L-iduronidase enzyme activity as measured in fibroblasts or leukocytes; OR
   • Documented alpha-L-iduronidase gene mutation.

Continuation requests for Aldurazyme (laronidase) may be approved if the following criterion is met:

1. 2. Documentation is provided that there is clinically significant improvement or stabilization in clinical signs and symptoms of disease (including but not limited to reduction in urinary GAG excretion, reduction in hepatosplenomegaly, improvement in pulmonary function, improvement in walking distance and/or improvement in fine or gross motor function) compared to the predicted natural history trajectory of disease.

   Requests for Aldurazyme (laronidase) may not be approved for the following:

   • Individual with the Scheie syndrome of MPS I with mild symptoms; OR
   • May not be approved when the above criteria are not met and for all other indications.

   Quantity Limits

   Aldurazyme (laronidase) Quantity Limit

   Drug	Limit
   Aldurazyme (laronidase) 2.9 mg vial	0.58 mg/kg once a week

   Coding

   The following codes for treatments and procedures applicable to this document are included below for informational purposes. Inclusion or exclusion of a procedure, diagnosis or device code(s) does not constitute or imply member coverage or provider reimbursement policy. Please refer to the member's contract benefits in effect at the time of service to determine coverage or non-coverage of these services as it applies to an individual member.

   HCPCS

   • J1931 - Injection, laronidase, 0.1 mg (Aldurazyme)
   • S9357 - Home infusion therapy, enzyme replacement intravenous therapy, (e.g., Imiglucerase); administrative services, professional pharmacy services, care coordination, and all necessary supplies and equipment (drugs and nursing visits coded separately), per diem

   ICD-10 Diagnosis

   • E76.01 - Hurler’s syndrome
   • E76.02 - Hurler-Scheie syndrome
   • E76.03 - Scheie’s syndrome
   • E76.3 - Mucopolysaccharidosis, unspecified

   Document History

   Revised: 9/11/2023