ALDURAZYME (laronidase)

Office-Administration – intravenous (IV) infusion

 

Diagnosis considered for coverage:

 

  • Mucopolysaccharidosis I (MPS I): Indicated for 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 risks and benefits of treating mildly affected patients with the Scheie form have not been established. Aldurazyme has not been evaluated for effects of the central nervous system manifestations of the disorder.

 

Coverage Criteria:

 

For diagnosis of Mucopolysaccharidosis I (MPS I):

  • One of the following (documented in medical records):
    • Diagnosis of Hurler or Hurler-Scheie form of MPS I
    • Diagnosis of Scheie form of MPS I in patients with moderate to severe symptoms.

 

Dosing:

 

  • The recommended dosage regimen of ALDURAZYME is 0.58 mg/kg of body weight administered once weekly as an intravenous infusion.
  • Each vial of ALDURAZYME provides 2.9 milligrams (mg) of laronidase in 5.0 milliliters (mL) of solution and is intended for single dose only.
  • Patient’s weight (kg) × 1 mL/kg of ALDURAZYME = Total number of mL of ALDURAZYME Total number of mL of ALDURAZYME ÷ 5 mL per Vial = Total number of Vials.

 

Coverage Duration:

 

  • One year

 

Authorization is not covered for the following:

The use of this drug for indications not listed in this policy does not meet the coverage criteria established by the Western Health Advantage (WHA) Pharmacy and Therapeutics (P&T) Committee.

 

Additional Information:
  • Mucopolysaccharide storage disorders are caused by the deficiency of specific lysosomal enzymes required for the catabolism of glycosaminoglycans (GAGs).
  • Mucopolysaccharidosis I (MPS I) is an autosomal recessive, progressive, life-threatening disorder characterized by the deficiency of α-L-iduronidase (IDUA), an enzyme needed for the degradation of the GAGs, heparan sulfate and dermatan sulfate. MPS I is phenotypically heterogeneous, with a diverse constellation of symptoms covering a broad spectrum of severity
  • Biochemical and clinical features of MPS I:
    • Hurler (severe): Usual diagnosis at age 1 to 2 years. Key distinct findings include developmental delay, severe coarse facies, hepatosplenomegaly, airway obstruction, dysostosis multiplex, death by age 10 years.
    • Hurler-Scheie (intermediate): Usual diagnosis at age 1 to 5 years. Key distinct findings include micrognathia, toe walking, moderate coarse facies, possible normal intelligence, death by 20s.
    • Scheie (least severe form): Usual diagnosis at age 3 to 15 years. Key distinct findings include aortic valve abnormalities, joint disease, corneal clouding, normal facies, death in decades.
  • The goals of MPS I treatment are to improve quality of life, slow down the progression of the disease, and prevent permanent tissue and organ damage. There is currently no cure for MPS I; however, early intervention may help prevent irreversible damage.
  • Most therapies for MPS I are directed toward treatment of complications and are not specific for the underlying abnormality. Supportive or symptomatic management can improve quality of life but cannot prevent the decline in function. Enzyme replacement (ERT) with Aldurazyme (laronidase) or hematopoietic stem cell transplantation (HSCT) may alter the natural history of these disorders. The choice of therapy depends upon the type of MPS and the disease severity.
  • Laronidase has a boxed warning for anaphylaxis and warnings for risks of acute respiratory complications, acute respiratory failure, and infusion reactions.

 

Policy Updates:
  • 03/01/2015 – New utilization management policy approved by WHA P&T (02/15/2023).

 

References:
  1. Aldurazyme Prescribing Information, BioMarin Pharmaceutical Inc. Novato, CA. December 2019.
  2. British Inherited Metabolic Diseases Group (BIMDG) Web site. Guidance for the treatment of MPS I. January 2020. https://bimdg.org.uk/store/lsd//MPS1Guide_LSDSS_Jan2020_340418_11032020.pdf.  Accessed December 29, 2022.
  3. Clarke LA, Wraith JE, Beck M, et al. Long-term efficacy and safety of laronidase in the treatment of mucopolysaccharidosis I. Pediatrics. 2009;123(1):229-240.
  4. Jameson E, Jones S, Remmington T. Enzyme replacement therapy with laronidase (Aldurazyme) for treating mucopolysaccharidosis type I. Cochrane Database Syst Rev. 2019;6(6):CD009354.
  5. Muenzer J, Wraith JE, Clarke LA; International Consensus Panel on Management and Treatment of Mucopolysaccharidosis I. Mucopolysaccharidosis I: management and treatment guidelines. Pediatrics. 2009;123(1):19-29.
  6. Sifuentes M, Doroshow R, Hoft R, et al. A follow-up study of MPS I patients treated with laronidase enzyme replacement therapy for 6 years. Mol Genet Metab. 2007;90(2):171-180.
  7. 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(5):581-588.

 

Last review date: March 1, 2023