CEREDASE SUMMARY
Ceredase® (alglucerase injection) is a modified form of the enzyme,ß-glucocerebrosidase (ß-D-glucosyl-N-acylsphingosine glucohydrolase, EC 3.2.1.45). Alglucerase is a monomeric glycoprotein of 497 amino acids with carbohydrates making up approximately 6% of the molecule (Mr=59,300 as determined by SDS-PAGE). The unmodified enzyme (ß-glucocerebrosidase) also contains 497 amino acids and contains approximately 12% carbohydrate (Mr=67,000). The carbohydrates on the unmodified enzyme consist of N-linked carbohydrate chains of the complex and high mannose type. Glucocerebrosidase and alglucerase catalyze the hydrolysis of the glycolipid, glucocerebrosidase, within the lysosomes of the reticuloendothelial system.
Ceredase® (alglucerase injection) is indicated for use as a long-term enzyme replacement therapy for children, adolescents and adult patients with a confirmed diagnosis of Type I Gaucher disease who exhibit signs and symptoms that are severe enough to result in one or more of the following conditions:
- moderate-to-severe anemia;
- thrombocytopenia with bleeding tendency;
- bone disease;
- significant hepatomegaly or splenomegaly.
|
NEWS HIGHLIGHTS
Published Studies Related to Ceredase (Alglucerase)
Dose-response relationships for enzyme replacement therapy with imiglucerase/alglucerase in patients with Gaucher disease type 1. [2009.02]
PURPOSE: To determine whether enzyme therapy with imiglucerase/alglucerase demonstrates dose-response relationships with doses and disease parameters used in routine clinical practice for Gaucher disease type 1 patients... CONCLUSIONS: Enzyme therapy with imiglucerase/alglucerase displays a dose-dependent improvement in hematological and visceral parameters in Gaucher disease type 1 patients. Group C displayed greater treatment effects than Groups A or B. Propensity score matching and nonlinear mixed effects model analyses provide a prototype for assessment of treatment outcomes based on observational data from international rare disease registries.
Mutation analysis of the acid beta-glucosidase gene in a patient with type 3 Gaucher disease and neutralizing antibody to alglucerase. [2001.11.01]
The beneficial effects of macrophage-targeted glucocerebrosidase (alglucerase, Ceredase) in patients with Gaucher disease are well established.Our results may help to shed light on the mechanisms underlying this phenomenon which, in the rare instances where it occurs, hampers the efficacy of enzyme replacement therapy.
Anaphylactoid reaction to imiglucerase, but not to alglucerase, in a type I Gaucher patient. [1999.04]
Imiglucerase, the recombinantly produced enzyme, is gradually replacing the human placental derived alglucerase in the treatment of gaucher patients. We describe the first case, to the best of our knowledge, of an anaphylactoid reaction to imiglucerase in a patient who tolerated alglucerase.This sensitivity to imiglucerase but not to alglucerase, raises the question of future treatment for this patient, since the production of alglucerase may cease, once imiglucerase production will cover the need for replacement enzyme.
Immunosurveillance of alglucerase enzyme therapy for Gaucher patients: induction of humoral tolerance in seroconverted patients after repeat administration. [1999.03.15]
Alglucerase, a macrophage-targeted enzyme replacement therapy for Gaucher disease, has been successfully used for several years to improve clinical symptoms and reverse disease progression. As part of an immunosurveillance program, 1,122 Gaucher patients were monitored for antibody response to glucocerebrosidase, the active component of alglucerase.
[Uveitis masquerade syndrome in Gaucher disease. Causal treatment by alglucerase substitution therapy] [1998.12]
CONCLUSIONS: The dramatic improvement occurred under treatment with alglucerase after no response to steroid treatment. Thus intermediate uveitis with severe vitritis can be interpreted as uveitis masquerade syndrome with M. Gaucher. To the best of our knowledge, this is the first case of M. Gaucher presenting initially as intermediate uveitis and showing successful clinical improvement during administration of alglucerase.
Clinical Trials Related to Ceredase (Alglucerase)
Safety and Efficacy of Cerezyme® Infusions Every 4 Weeks Versus Every 2 Weeks in Type 1 Gaucher Disease [Completed]
This is a multicenter, randomized trial to compare the safety and efficacy of two dosing
frequencies of Cerezyme® in patients with Gaucher disease who are currently being treated
with Cerezyme®.
Approximately 90 patients will be randomized in a 2: 1 (q4 : q2) ratio to one of two treatment
arms at up to 26 study centers worldwide. Patients will continue to receive the same total
4-week dose that they were receiving prior to study enrollment, however, they will be
randomized to receive either their total 4-week dose in two infusions, one infusion every 2
weeks or their total 4-week dose in one infusion every 4 weeks. The randomization scheme
will ensure a 2: 1 balance between the every 4-week versus every 2-week infusion groups,
respectively.
Study of GA-GCB Enzyme Replacement Therapy in Type 1 Gaucher Disease Patients Previously Treated With Imiglucerase [Recruiting]
Gaucher disease is a rare lysosomal storage disorder caused by the deficiency of the enzyme
glucocerebrosidase (GCB). Due to the deficiency of functional GCB, glucocerebroside
accumulates within macrophages leading to cellular engorgement, organomegaly, and organ
system dysfunction. The purpose of this study is to evaluate the safety and efficacy of every
other week dosing of GA-GCB (velaglucerase alfa) in patients with type 1 Gaucher disease who
were previously treated with imiglucerase.
A Multicenter Study of the Efficacy of Cerezyme in Testing Skeletal Disease in Patients With Type I Gaucher Disease. [Completed]
This is a multicenter, open-label, prospective study of the efficacy of Cerezyme in treating
patients with skeletal manifestations secondary to Type I Gaucher disease.
The study objective is to evaluate and quantify skeletal responses as compared to baseline in
Type I gaucher disease patients receiving Cerezyme therapy for 48 months. Additional
objectives were to assess the usefulness of various skeletal parameters, such as bone pain,
bone crises, bone mineral density, and serum and urine bone markers, as indicative of
treatment response and may be useful in dose management.
Switchover Trial From Imiglucerase to Plant Cell Expressed Recombinant Human Glucocerebrosidase [Recruiting]
This is a multi-center, open-label, switchover trial to assess the safety of prGCD in 15
patients with Gaucher disease who are currently being treated with imiglucerase (Cerezyme®)
ERT.
A Study of Genz-112638 in Patients With Gaucher Disease Who Have Been Stabilized on Cerezyme [Recruiting]
This Phase 3 study was designed to confirm the efficacy and safety of Genz-112638 in
patients with Gaucher disease type 1 who have been stabilized on Cerezyme
|
|
|
|
Page last updated: 2009-10-20
|
