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THIS IS ONLY A MODEL LETTER AND SHOULD BE CUSTOMIZED TO ADDRESS
PATIENT-SPECIFIC ISSUES.
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[Date]
[Contact Name]
[Insurance Company]
[Street Address]
[City], [State] [Zip]
Patient Name: [Patient Name]
Subscriber ID#: [ID Number]
Group#: [Group Number]
Subject: Intent to Treat with Fabrazyme (agalsidase beta)
Dear [Contact Name]:
[Patient Name] [Patient ID Number] has been diagnosed with Fabry disease and I plan to treat [him/her]
with Fabrazyme (agalsidase beta), an enzyme replacement therapy. Fabrazyme is indicated for use in
patients with Fabry disease. It was approved by FDA on April 24, 2003. Fabrazyme is administered
intravenously and is typically administered on an outpatient basis. Prior to the availability of Fabrazyme,
treatment for Fabry disease was directed only at managing the patient’s symptoms and ameliorating the
life threatening complications. Fabrazyme is the only FDA-approved therapy for the treatment of this
life-threatening, orphan disease.
Fabry disease is a progressive, X-linked genetic disorder resulting from a defect in the gene for the
lysosomal enzyme, alpha-galactosidase A (-GAL). Partial or complete deficiency of -GAL leads to an
accumulation of lipids, particularly globotriaosylceramide (GL-3), in tissues throughout the body. The
inability to catabolize GL-3 may lead to progressive and often irreversible damage to the kidney, heart
and cerebrovascular system. The clinical course of Fabry disease is often marked by chronic pain,
angiokeratomas, hypohidrosis, heat and cold intolerance, corneal opacities, renal failure, stroke, and
cardiac complications. As the disease progresses, complications frequently become life threatening.
Produced by recombinant DNA technology, Fabrazyme has the same amino acid sequence as the native
enzyme. Fabrazyme replaces the missing enzyme and works by clearing the fatty substances that
accumulate in certain cells and tissues of Fabry patients. Fabrazyme reduces GL-3 deposition from the
capillary endothelium of the kidney and certain other cell types. Reduction to normal or near normal
levels has also been demonstrated in other renal cell types, such as mesangial cells, glomerular capillary
endothelium, interstitial cells and non-capillary endothelium. The reduction of GL-3 inclusions suggests
that Fabrazyme may ameliorate disease expression of Fabry disease; however, the relationship of GL-3
inclusion reduction to specific clinical manifestations of Fabry disease has not been established.
The most serious and most common adverse reactions reported with Fabrazyme are infusion reactions.
Infusion reactions may include tachycardia, hypertension, throat tightness, chest pain/tightness, dyspnea,
fever, chills/rigors, abdominal pain, pruritus, urticaria, nausea, vomiting, lip or ear edema, rash,
hypotension, myalgia, and headache.
The diagnosis of Fabry disease is frequently delayed until adulthood when the disease has progressed to
major organ dysfunction or failure. The progressive nature of Fabry disease may result in a substantially
decreased life expectancy and is often attributed to events associated with cerebrovascular disease,
cardiovascular disease, and renal failure. Therefore, it is important to diagnosis and treat Fabry disease
before extensive and/or irreversible damage to affected tissues occurs. To this end, I feel it is medically
necessary to initiate Fabrazyme treatment for [Patient Name] as soon as possible.
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 Fabrazyme is medically
indicated and necessary for treatment of [his/her] Fabry disease. Initially, my prescribed dosing regimen
will be [Dose] mg per kilogram, administered every two weeks.
Action Requested
Please send verification of [Patient Name]’s coverage for enzyme replacement therapy with Fabrazyme
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
cc [Patient Name]