Fabrazyme

About Fabrazyme

• Fabrazyme is indicated for long-term enzyme replacement therapy in patients who are being diagnosed with α-galactosidase A deficiency which is called as Fabry disease.
• Chemically it is a hydrolytic lysosomal neutral glycosphingolipid-specific enzyme.
• Fabrazyme (Agalsidase beta) becomes an exogenous source of α-galactosidase A in Fabry disease patients. 
• Agalsidase beta is transported into lysosomes where it exerts enzymatic activity and reduces accumulated levels of globotriaosylceramide (GL-3). 

Strength: 

Fabrazyme is intended for injection use available in 5 mg and 35 mg lyophilized cake or powder in a single-dose vial for reconstitution.

Recommended Dosage:

• Fabrazyme dose is 1 mg/kg body weight given every two weeks as an intravenous injection.
• Appropriate medical support should be available while Fabrazyme is administered because of the potential for anaphylaxis and severe infusion-associated reactions. 
• Antipyretics to be given prior to infusion.
• The initial intravenous infusion rate is 0.25 mg/min (15 mg/hour). It is slowed down in the event of infusion-associated reactions.
• For patients of weight >30 kg, after tolerance to the infusion is established, increase the infusion rate in increments of 0.05 to 0.08 mg/min with each subsequent infusion. The minimum infusion duration is 1.5 hours.
• For patients weighing <30 kg, the maximum infusion rate is 0.25 mg/minute (15 mg/hour).
• Once a patient tolerates the infusion, the dose may be increased to reach the dose of 1 mg/kg and the infusion rate may be increased by slowly titrating and 30 minutes up to a maximum rate of 0.25 mg/minute), as per the tolerance.

Warnings & Precautions

Anaphylaxis and Hypersensitivity Reactions: If severe hypersensitivity or anaphylactic reactions occur, immediately infusion should be discontinued the infusion. Infusion should be performed under the supervision of a qualified professional. For infusion-associated reactions, decrease the infusion rate or stop temporarily and consider administration of other antipyretics and antihistamines. In case of severe infusion-associated reaction discontinue the infusion and initiate appropriate anaphylaxis treatment.

Common Fabrazyme Side Effects

• Upper respiratory tract infection
• Chills
• Pyrexia
• Headache
• Cough
• Paresthesia
• Fatigue
• Peripheral edema
• Dizziness
• Rash

Use in Specific Population

Pregnancy: No data identified a drug-associated risk of major birth defects, miscarriage or adverse maternal or fetal events.

Pediatric use:  The safety and effectiveness of Fabrazyme is established in pediatric patients based on a well-controlled study.

Storage and Handling

• Fabrazyme (Agalsidase beta) for injection is supplied in a sterile, nonpyrogenic, white to off-white lyophilized cake or powder in single-dose vials.
• Fabrazyme vials should be refrigerated at 2°C to 8°C (36°F to 46°F). Do not use Fabrazyme after the expiry date.
• Reconstituted and diluted Fabrazyme should be used immediately. If not used immediately, then solution may be stored for up to 24 hours at 2°C to 8°C.