If you have a patient with Fabry disease, are seeking information on treatment with Fabrazyme® (agalsidase beta), or think you may have an undiagnosed patient and want information on signs and symptoms and diagnosis, this section has information and resources that may help:
Enhance recognition of signs and symptoms
Facilitate diagnosis of patients and their family members
Explain treatment with Fabrazyme
Facilitate discussions with your patients about the disorder and treatment options
Diagnostic Testing Early
diagnosis of patients with Fabry disease is important. Diagnosis can be confirmed biochemically by demonstration
of very low or undetectable α-GAL activity in plasma, serum, leukocytes, tears, or biopsied tissues.
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About Treatment For more about Fabrazyme,
and the infusion process, visit About Fabrazyme.
Fabrazyme (agalsidase beta) is indicated for use in patients with Fabry
disease. Fabrazyme reduces globotriaosylceramide (GL-3) deposition in
capillary endothelium of the kidney and certain other cell types. The
reduction of GL-3 inclusions suggests that Fabrazyme may ameliorate disease
expression; 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. Serious and/or frequently occurring related adverse
reactions consisted of one or more of the following events: chills, pyrexia,
feeling hot or cold, dyspnea, nausea, flushing, headache, vomiting,
paresthesia, fatigue, pruritus, pain in extremity, hypertension, chest pain,
throat tightness, abdominal pain, dizziness, tachycardia, nasal congestion,
diarrhea, edema peripheral, myalgia, back pain, pallor, bradycardia,
urticaria, hypotension, face edema, rash, and somnolence. The occurrence of
somnolence can be attributed to clinical trial specified pre-treatment with
antihistamines.
Other reported serious adverse events included stroke, pain, ataxia,
bradycardia, cardiac arrhythmia, cardiac arrest, decreased cardiac output,
vertigo, hypoacousia, and nephrotic syndrome. These adverse events also
occur as manifestations of Fabry disease; an alteration in frequency or
severity cannot be determined from the small numbers of patients studied.
Infusion reactions occurred in many patients treated with Fabrazyme and some
of the reactions were severe. Patients should be given antipyretics prior
to infusion. Infusion reactions occurred in some patients after receiving
pretreatment with antipyretics, antihistamines, and oral steroids. Infusion
reactions declined in frequency with continued use of Fabrazyme. However,
infusion reactions may still occur despite extended duration of Fabrazyme
treatment. Because of the potential for severe infusion reactions,
appropriate medical support measures should be readily available when
Fabrazyme is administered.
Patients with compromised cardiac function should be monitored closely if
the decision is made to administer Fabrazyme.
Most patients develop IgG antibodies to Fabrazyme. A few patients developed
IgE or skin test reactivity specific to Fabrazyme. Physicians should
consider testing for IgE in patients who experienced suspected allergic
reactions and consider the risks and benefits of continued treatment in
patients with anti- Fabrazyme IgE. Patients with Fabrazyme- specific IgE
antibody have been treated using a rechallenge protocol. Rechallenge of
these patients should only occur under the direct supervision of qualified
personnel, with appropriate medical support measures readily available.
The safety and efficacy in patients younger than 8 years of age have not
been evaluated. IgE immunologic responses in pediatric patients may differ
from those in adults, as IgG seroconversion was associated with prolonged
half-life concentrations of Fabrazyme, which is rarely observed in adult
patients.
Fabrazyme is available by prescription only. Side effects should be reported
promptly to Genzyme Medical Information at 800-745-4447, option 2. To learn
more, please see the full
prescribing information (PDF) or contact Genzyme at 1-800-745-4447.
Looking to refer a patient? There are treatment centers around the country with experience in the care of patients with Fabry disease. More >
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