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THIS IS A MODEL LETTER - PLEASE CUSTOMIZE FOR YOUR PATIENT
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[Date]
[Contact Name]
[Insurance Company]
[Street Address]
[City], [State] [Zip]
Patient Name:
Subscriber ID#:
Group#:
[Patient Name]
[ID Number]
[Group Number]
Subject: Intent to Treat with Aldurazyme (laronidase)
Dear [Contact Name]:
I am writing on behalf of my patient, [Patient Name,] who has been diagnosed with Mucopolysaccharidosis I disease
(MPS I) and for whom I plan to treat with Aldurazyme® (laronidase) , an enzyme replacement therapy. Aldurazyme
is indicated for patients with Hurler and Hurler-Scheie forms of MPS I and for Scheie patients with moderate to
severe symptoms. The risks and benefits of treating mildly affected patients with the Scheie form have not been
established. Prior to the availability of Aldurazyme, treatment for MPS I was directed at managing the patient’s
symptoms and ameliorating the life threatening complications. Aldurazyme is the only enzyme replacement therapy
available that treats the underlying cause of MPS I. Aldurazyme has been shown to improve pulmonary function,
and walking capacity as well as decrease glycosaminoglycans (GAG, the accumulated substrate), and reduce
hepatomegaly in patients with this disorder. The therapy has not been evaluated for effects on the central nervous
system manifestations of this disease. Aldurazyme is administered intravenously and is typically administered on an
outpatient basis.
MPS I is a progressive, autosomal recessive genetic disorder resulting from a defect in the gene for the lysosomal
enzyme -L-iduronidase. Partial or complete deficiency of -L-iduronidase leads to progressive accumulation of
glycosaminoglycans dermatan sulfate and heparan sulfate, which are complex polysaccharides that are an important
component of the extracellular matrix and connective tissues throughout the body. Deficiency of this enzyme leads
to accumulation of GAG in the lysosome, ultimately resulting in cell, tissue and organ dysfunction. The
heterogeneity of MPS I disease demonstrates a wide range of clinical involvement marked by umbilical and inguinal
hernias, skeletal abnormalities, recurrent and persistent upper respiratory tract infections, coarse facial features,
arthropathy, hydrocephalus, spinal root and peripheral nerve entrapment, obstructive airway disease, sleep apnea,
hearing loss, massive hepatosplenomegaly, corneal clouding, glaucoma, retinal degeneration, optic atrophy, cardiac
valvular and ischemic myocardial damage. As MPS I progresses, complications become debilitating and often life
threatening.
The most common adverse reactions associated with Aldurazyme treatment in the clinical studies were upper
respiratory tract infection, rash, and injection site reaction. The most common adverse reactions requiring
intervention were infusion-related reactions, including flushing, fever, headache, and rash. The most serious adverse
reaction reported with Aldurazyme was an anaphylactic reaction, which occurred in 1 patient approximately 3 hours
after the start of the infusion. The reaction consisted of urticaria and airway obstruction. Resuscitation required an
emergency tracheotomy. This patient’s pre-existing MPS I-related upper airway obstruction may have contributed to
the severity of this reaction. Approximately 91% of patients treated with Aldurazyme were positive for antibodies to
laronidase. The clinical significance of antibodies to Aldurazyme® (laronidase) is not known, including the potential
for product neutralization.
There are no known contraindications to the use of Aldurazyme. Adverse reactions should be reported promptly to
Genzyme Corporation at 1-800-745-4447. Aldurazyme is available by prescription only. For more information on
Aldurazyme therapy, please see full prescribing information, visit www.aldurazyme.com or contact Genzyme at 1800-745-4447.
Documentation Enclosed
The attached Statement of Medical Necessity contains information pertaining to [Patient Name]’s clinical history,
diagnosis and signs and symptoms - demonstrating that the use of Aldurazyme is medically indicated for treatment of
[his/her] MPS I disease. Initially, my prescribed dosing regimen will be
[
] mg per kilogram administered [
] per week.
Action Requested
Please send verification of [Patient Name]’s coverage for enzyme replacement therapy with Aldurazyme as soon as
possible. If you have any questions pertaining to [Patient Name]’s clinical history and/or my treatment plan, please
call me at [Phone Number].
Thank you for your immediate attention to this request.
Sincerely,
[Physician Name]
Enclosure: Statement of Medical Necessity
cc [Patient Name/Legal Guardian]
AZ/US/P035A/05/07