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.
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NEWS HIGHLIGHTS
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.
Conditional modeling of antibody titers using a zero-inflated poisson random effects model: application to Fabrazyme((R)). [2009.09.30]
Patients that are exposed to biotechnology-derived therapeutics often develop antibodies to the therapeutic, the magnitude of which is assessed by measuring antibody titers...
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 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 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. Fabrazyme is a drug that helps to breakdown
and removes 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
(globatriaosylceramide or GL-3) levels in these tissues in particular is thought to cause the
clinical symptoms that are common to Fabry disease. This study will test the safety and
efficacy of Fabrazyme in the treatment of patients with Fabry disease.
A Study of the Safety and Efficacy of Fabrazyme as Compared to Placebo in Patients With Advanced Fabry Disease [Completed]
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
("globatriaosylceramide" or "GL-3") levels in these tissues in particular is thought to cause
the clinical symptoms that are common to Fabry disease. This study will test the safety and
efficacy of Fabrazyme in the treatment of patients with Fabry disease.
A Study of Fabrazyme in Pediatric Patients With Fabry Disease [Completed]
People with Fabry disease have an alteration in their genetic material (DNA) which causes a
deficiency of the a-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 a-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 study will explore the safety and pharmacokinetics of
Fabrazyme in pediatric patients aged between 7 and 15 years.
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Page last updated: 2009-10-20
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