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Pronunciation:

(aye GAL si days BAY ta)

U.S. Brand Names:

Fabrazyme®

Synonyms:

Alpha-Galactosidase-A (Human, Recombinant); r-h -GAL

Generic Available:

Use:

Replacement therapy for Fabry disease

Pregnancy Risk Factor:

Pregnancy Implications:

There are no adequate and well-controlled studies in pregnant women. Women of childbearing potential are encouraged to enroll in Fabry registry.

Lactation:

Excretion in breast milk unknown/use caution

Contraindications:

No known contraindications

Warnings/Precautions:

Infusion reactions are common, and may be severe; pretreatment with antipyretics is advised. Use caution in cardiovascular disease (risk related to infusion reactions may be increased). A registry has been created to monitor therapeutic responses and adverse effects during long-term treatment; patients should be encouraged to register (www.fabryregistry.com or 1-800-745-4447). Safety and efficacy in pediatric patients have not been established (studies limited to patients 16 years of age).

Adverse Reactions:

Note: The most common and serious adverse reactions are infusion reactions (symptoms may include fever, tachycardia, hypertension, throat tightness, dyspnea, chills, abdominal pain, pruritus, urticaria, vomiting).

>10%:

Cardiovascular: Edema (21%), chest pain (17%), hypotension (14%)

Central nervous system: Fever (48%), headache (45%), anxiety (28%), pain (21%), dizziness (14%), paresthesia (14%)

Dermatologic: Pallor (14%)

Gastrointestinal: Nausea (28%)

Miscellaneous: Infusion reactions (alteration of temperature sensation 17%)

Neuromuscular & skeletal: Rigors (52%), skeletal pain (21%)

Respiratory: Rhinitis (38%), pharyngitis (28%)

1% to 10%:

Cardiovascular: Cardiomegaly (10%), hypertension (10%)

Central nervous system: Depression (10%)

Gastrointestinal: Dyspepsia (10%)

Genitourinary: Testicular pain (7%)

Neuromuscular & skeletal: Arthrosis (10%)

Respiratory: Bronchitis (10%), bronchospasm (7%), laryngitis (7%), sinusitis (7%)

Other reported severe reactions (frequency not established): Arrhythmia, ataxia, bradycardia, cardiac arrest, cardiac output decreased, nephritic syndrome, stroke, vertigo

Drug Interactions:

No formal drug interaction studies have been conducted.

Stability:

Store vials between 2°C and 8 °C (36°F and 46°F). Final infusion should be used immediately if possible, but may be stored for up to 24 hours between 2°C and 8 °C (36°F and 46°F). Each vial should be reconstituted with 7.2 mL SWFI; inject down internal side wall of vial; roll and tilt gently. Resulting solution contains 5 mg/mL. To make final infusion, add the desired amount of reconstituted solution (based on patient weight) to make a final volume of 500 mL in NS (do not use filter needle to prepare). Avoid vigorous shaking or agitation.

Compatibility:

Compatible in NS. Should not be infused with other products.

Mechanism of Action:

Agalsidase beta is a recombinant form of the enzyme alpha-galactosidase-A, which is required for the hydrolysis of GL-3 and other glycosphingolipids. The compounds may accumulate (over many years) within the tissues of patients with Fabry disease, leading to renal and cardiovascular complications. In clinical trials of limited duration, agalsidase been noted to reduce tissue inclusions of a key sphingolipid (GL-3). It is believed that long-term enzyme replacement may reduce clinical manifestations of renal failure, cardiomyopathy, and stroke. However, the relationship to a reduction in clinical manifestations has not been established.

Pharmacodynamics/Kinetics:

Half-life elimination: 42-102 minutes (nonlinear)

Dosage:

I.V.: Adults: 1 mg/kg every 2 weeks

Dosage adjustment in renal impairment: No dosage adjustment required

Administration:

Antipyretics should be administered prior to infusion. Initial infusion rate should not exceed 0.25 mg/minute (15 mg/hour). Decrease rate in the event of an infusion reaction. After patient tolerance to the infusion is established, rate may be increased in increments of 0.05-0.08 mg/minute (3-5 mg/hour) with each subsequent infusion. A 0.2 micron low protein-binding filter may be used.

Monitoring Parameters:

Development of IgG or IgE antibodies in patients with suspected allergic reactions (test available from manufacturer).

Patient Education:

This medication will not cure Fabry's Disease, rather it may help to reduce or slow the progression of symptoms. This medication can only be administered by infusion. You will be monitored during and after each dose. Report immediately any tightness in your throat or chest, difficulty breathing; chills, fever, vomiting; or acute headache at the time of infusion. Report any chest pain; swelling of extremities; headache, nausea, or vomiting; skeletal pain; infusion site redness or swelling; or other adverse reactions that occur between doses. Pregnancy/breast-feeding precautions: Notify prescriber if you are or intend to become pregnant. Consult prescriber if breast-feeding.

Dental Health: Effects on Dental Treatment:

No significant effects or complications reported

Dental Health: Vasoconstrictor/Local Anesthetic Precautions:

No information available to require special precautions

Mental Health: Effects on Mental Status:

Anxiety and dizziness are common; may cause depression

Mental Health: Effects on Psychiatric Treatment:

None reported

Dosage Forms:

Injection, powder for reconstitution: 35 mg [contains mannitol 222 mg/vial; derived from Chinese hamster cells]

International Brand Names:

Fabrazyme® (AT, CH, DE, DK, ES, FR, IL, IT, NO, PL, SE)

References

Eng C, Guffton N, Wilcox WR, et al, "Safety and Efficacy of Recombinant Human Alpha-Galactosidase A Replacement Therapy in Fabry's Disease,"N Engl J Med, 2001, 345(1):9-16.

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