NEWS HIGHLIGHTSMedia Articles Related to Cerezyme (Imiglucerase)
Elelyso Approved for Gaucher Disease
Source: MedicineNet Gaucher Disease Specialty [2012.05.02]
Title: Elelyso Approved for Gaucher Disease
Category: Health News
Created: 5/1/2012 8:05:00 PM
Last Editorial Review: 5/2/2012 12:00:00 AM
Published Studies Related to Cerezyme (Imiglucerase)
A randomized trial comparing the efficacy and safety of imiglucerase (Cerezyme) infusions every 4 weeks versus every 2 weeks in the maintenance therapy of adult patients with Gaucher disease type 1. [2009.04]
Imiglucerase (Cerezyme) has been the standard of care for treatment of Gaucher disease, a lysosomal storage disorder resulting from deficiency of glucocerebrosidase, since its approval in 1994. Infusions are typically given once every 2 weeks... Continued monitoring in patients treated with Q4 dosing is required to assess long-term effectiveness.
Low frequency maintenance therapy with imiglucerase in adult type I Gaucher disease: a prospective randomized controlled trial. [2007.02]
BACKGROUND AND OBJECTIVES: Gaucher disease type I can be successfully treated with enzyme replacement therapy (ERT). In order to reduce the burden of the intravenously administered enzyme, a low frequency of administration was prospectively studied in patients with stable and minor disease following ERT... CONCLUSIONS: Low frequency ERT in adult Gaucher type I patients maintains stable disease in most, but not all patients with stable and minimal disease. Close monitoring of all disease parameters remains mandatory.
Osteopenia in Gaucher disease develops early in life: response to imiglucerase enzyme therapy in children, adolescents and adults. [2011.01.15]
BACKGROUND: In Gaucher disease (GD), acid-beta-glucosidase (GBA1) gene mutations result in defective glucocerebrosidase and variable combinations of hematological, visceral, and diverse bone disease. Osteopenia is highly prevalent, but its age of onset during the natural course of GD is not known. It is also unclear if the degree of improvement in osteopenia, secondary to imiglucerase enzyme therapy, differs by the age of the patient. OBJECTIVE: We hypothesized that osteopenia develops early in life, during the natural course of type 1 Gaucher disease (GD1), and that its response to treatment is maximal during this period... CONCLUSIONS: Low bone density is common in GD1 with the highest prevalence rate in adolescence, a developmental period critical to attainment of peak bone mass. Imiglucerase results in amelioration of osteopenia in all age groups, with the greatest improvements in younger patients. Copyright (c) 2010 Elsevier Inc. All rights reserved.
Evaluation of Spanish Gaucher disease patients after a 6-month imiglucerase shortage. [2011.01.15]
Recently, an acute restriction of imiglucerase has occurred as a result of viral contamination and manufacturing problems... No significant changes in blood counts, visceral volumes and CCL18/PARC concentration were observed.
Nonprecipitous changes upon withdrawal from imiglucerase for Gaucher disease because of a shortage in supply. [2011.01.15]
Because of an unpredicted and unavoidable shortage in global supplies of imiglucerase for Gaucher disease, we collected clinical and laboratory data from patients who were evaluated </= 6months before drug withdrawal and then retested before/during reinstatement of therapy... Although we no longer think that drug vacations should be considered as a form of maintenance for patients requiring enzyme therapy for Gaucher disease, but if necessary-for compelling personal reasons-one may be reassured that a short-term drug interruption is not likely to lead to irreversible complications or return to baseline values of critical clinical parameters.
Clinical Trials Related to Cerezyme (Imiglucerase)
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.
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 taliglucerase
alfa in 30 patients with Gaucher disease who are currently being treated with imiglucerase
(Cerezyme®) ERT.
Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) ERT Compared With Imiglucerase in Type I Gaucher Disease [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 efficacy and safety of
GA-GCB (velaglucerase alfa) administered every other week in comparison to imiglucerase in
treatment naive patients with type 1 Gaucher disease.
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.
Reports of Suspected Cerezyme (Imiglucerase) Side Effects
Death (23),
Disease Progression (21),
Convulsion (17),
Pyrexia (14),
Gaucher's Disease (14),
Fall (11),
Abortion Spontaneous (10),
Tremor (9),
Headache (8),
Thrombocytopenia (8), more >>
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