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Fabrazyme (Agalsidase Beta) - Summary

 
 



FABRAZYME SUMMARY

Fabrazyme® is a recombinant human (alpha)-galactosidase A enzyme with the same amino acid sequence as the native enzyme.

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.


See all Fabrazyme indications & dosage >>

NEWS HIGHLIGHTS

Media Articles Related to Fabrazyme (Agalsidase Beta)

Fabry Disease
Source: MedicineNet carbamazepine Specialty [2014.03.26]
Title: Fabry Disease
Category: Diseases and Conditions
Created: 12/31/1997 12:00:00 AM
Last Editorial Review: 3/26/2014 12:00:00 AM

more news >>

Published Studies Related to Fabrazyme (Agalsidase Beta)

A retrospective analysis of the potential impact of IgG antibodies to agalsidase beta on efficacy during enzyme replacement therapy for Fabry disease. [2009.01]
Fabry disease results from a genetic deficiency of alpha-galactosidase A (alpha GAL) and the impaired catabolism of globotriasoylceramide (GL-3) and other glycosphingolipid substrates, which then accumulate pathogenically within most cells. Enzyme replacement therapy (ERT) with agalsidase beta (Fabrazyme), one of two available forms of recombinant human alpha GAL, involves regular intravenous infusions of the therapeutic protein...

Treatment of Fabry disease: outcome of a comparative trial with agalsidase alfa or beta at a dose of 0.2 mg/kg. [2007.07.11]
CONCLUSION: Our study revealed no difference in reduction of left ventricular mass or other disease parameters after 12 and 24 months of treatment with either agalsidase alfa or beta at a dose of 0.2 mg/kg biweekly. Treatment failure occurred frequently in both groups and seems related to age and severe pre-treatment disease. TRIAL REGISTRATION: International Standard Randomized Clinical Trial ISRCTN45178534 [http://www.controlled-trials.com/ISRCTN45178534].

Sustained, long-term renal stabilization after 54 months of agalsidase Beta therapy in patients with fabry disease. [2007.05]
Fabry disease, an inherited deficiency of the lysosomal enzyme alpha-galactosidase A, causes progressive intralysosomal accumulation of globotriaosylceramide (GL-3) and premature death from renal, cardiac, and cerebrovascular manifestations. To determine the long-term safety and efficacy of recombinant human alpha-galactosidase A, an open-label, phase III extension study was conducted, involving 58 patients who had classic Fabry disease and completed a 20-wk, double-blind, randomized, placebo-controlled, phase III study of agalsidase beta and were transitioned to an extension trial to receive biweekly 1 mg/kg agalsidase beta for up to an additional 54 mo...

Agalsidase-beta therapy for advanced Fabry disease: a randomized trial. [2007.01.16]
BACKGROUND: Fabry disease (alpha-galactosidase A deficiency) is a rare, X-linked lysosomal storage disorder that can cause early death from renal, cardiac, and cerebrovascular involvement. OBJECTIVE: To see whether agalsidase beta delays the onset of a composite clinical outcome of renal, cardiovascular, and cerebrovascular events and death in patients with advanced Fabry disease... CONCLUSIONS: Agalsidase-beta therapy slowed progression to the composite clinical outcome of renal, cardiac, and cerebrovascular complications and death compared with placebo in patients with advanced Fabry disease. Therapeutic intervention before irreversible organ damage may provide greater clinical benefit.

Update on role of agalsidase alfa in management of Fabry disease. [2011.03.14]
Fabry disease (FD) is an X-linked lysosomal storage disorder that affects both men and women.This update focuses on published data on the efficacy and tolerability of enzyme replacement therapy with agalsidase alfa, and gives a brief overview on some of the outstanding management issues in the treatment of this complex disease.

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Clinical Trials Related to Fabrazyme (Agalsidase Beta)

A Study in Patients With Fabry Disease Who Are on Chronic Hemodialysis Therapy for Treatment of End-Stage Renal Insufficiency. [Terminated]
People with Fabry disease have an alteration in their genetic material (DNA) which causes a deficiency of the a-galactosidase A enzyme. Fabrazyme is a drug that helps to breakdown and remove certain types of fatty substances called “glycolipids.” These glycolipids are normally present within the body in most cells. In Fabry disease, glycolipids build up in various tissues such as the liver, kidney, skin, and blood vessels because a-galactosidase A is not present, or is present in small quantities. The build up of glycolipid (globotriaosylceramide or GL-3) levels in these tissues in particular is thought to cause the clinical symptoms that are common to Fabry disease. This study is designed to verify that no loss of Fabrazyme occurs during simultaneous Fabrazyme infusion and hemodialysis in patients currently receiving Fabrazyme at a dose of 1. 0 mg/kg every 2 weeks.

A Study of Two Fabrazyme (Agalsidase Beta) Dosing Regimens in Treatment-naïve, Male Pediatric Patients Without Severe Symptoms [Recruiting]
The purpose of this study is to determine whether 2 alternative dosing regimens of Fabrazyme (agalsidase beta) (1. 0 mg/kg every 4 weeks or 0. 5 mg/kg every 2 weeks) are effective in treatment-naïve pediatric patients without severe symptoms.

A Study of the Effects of Fabrazyme (Agalsidase Beta) on Mother's Lactation and on the Growth, Development and Immunologic Response of Their Infants [Recruiting]
The purpose of this study is to observe the potential effects of Fabrazyme (agalsidase beta) treatment on lactation and on the growth, development, and immunologic response of infants born to mothers with Fabry disease who are treated with Fabrazyme during lactation. There are 3 participation scenarios: mother/infant full participation, mother full participation/infant development assessment only, and mother full participation/infant no participation. Whether or not the mother continues to lactate will be assessed at each visit. If the mother is no longer lactating, the mother will discontinue this study but continue to be followed in the Fabry Registry. The infant (if participating) will be followed for development only for the remainder of this 24 month study.

A Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease [Completed]
People with Fabry disease have an alteration in their genetic material (DNA) which causes a deficiency of the alpha-galactosidase A enzyme. This enzyme helps to break down and remove certain types of fatty substances called "glycolipids." These glycolipids are normally present within the body in most cells. In people with Fabry disease, glycolipids build up in various tissues such as the liver, kidney, skin, and blood vessels because alpha-galactosidase A is not present, or is present in small quantities. The build up of glycolipid levels (also referred to as "globotriaosylceramide" or "GL-3") in these tissues is thought to cause the clinical symptoms that are common to Fabry disease. Symptoms commonly appear during childhood with pain in the hands and feet. This trial is designed to evaluate the efficacy of a lower dose of Fabrazyme in patients who initially received 1. 0 mg/kg every 2 weeks of Fabrazyme by investigating if the achieved clearance of glycosphingolipid deposits in the vascular endothelium of the kidney can be maintained at a lower dose.

A Long Term Safety and Efficacy Study of Fabrazyme Replacement Therapy in Japanese Patients With Fabry Disease. [Recruiting]
The purpose of this survey is to identify any concerns regarding the following efficacy and safety-related issues in clinical practice with the new drugs "Fabrazyme for intravenous infusion 5mg" and "Fabrazyme for intravenous infusion 35mg" and to confirm the safety of these products in long-term use in the clinical setting.

1. New adverse drug reactions (ADRs) that cannot be predicted from the Precautions (in particular, clinically significant ADRs)

2. The incidence of ADRs under the actual conditions of use of the drug

3. Causal factors that might potentially affect safety

4. Efficacy evaluation in long-term use

This survey will be conducted in accordance with the approval condition established for Fabrazyme:

"To conduct a special surveillance of Efficacy and Safety in long term treatment and Pediatric with the drug."

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Reports of Suspected Fabrazyme (Agalsidase Beta) Side Effects

Renal Failure (17)Dyspnoea (14)Pain (12)Pyrexia (12)Cerebrovascular Accident (12)Cardiac Disorder (11)Death (10)Cardiac Valve Disease (10)Vomiting (9)Deafness Unilateral (8)more >>


Page last updated: 2014-03-26

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