ALDURAZYME SUMMARY
ALDURAZYME® (laronidase) is a polymorphic variant of the human enzyme, (alpha)-L-iduronidase that is produced by recombinant DNA technology in a Chinese hamster ovary cell line. (alpha)-L-iduronidase (glycosaminoglycan (alpha)-L-iduronohydrolase, EC 3.2.1.76) is a lysosomal hydrolase that catalyses the hydrolysis of terminal (alpha)-L-iduronic acid residues of dermatan sulfate and heparan sulfate.
ALDURAZYME is indicated for patients with Hurler and Hurler-Scheie forms of Mucopolysaccharidosis I (MPS I) and for patients with the Scheie form who have moderate to severe symptoms. The risks and benefits of treating mildly affected patients with the Scheie form have not been established.
ALDURAZYME has been shown to improve pulmonary function and walking capacity. ALDURAZYME has not been evaluated for effects on the central nervous system manifestations of the disorder.
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NEWS HIGHLIGHTS
Published Studies Related to Aldurazyme (Laronidase)
A dose-optimization trial of laronidase (Aldurazyme) in patients with mucopolysaccharidosis I. [2009.01]
Recombinant human alpha-l-iduronidase (Aldurazyme), laronidase) is approved as an enzyme replacement therapy to treat the lysosomal storage disorder, mucopolysaccharidosis type I (MPS I) at a dose of 0.58 mg/kg by once-weekly intravenous infusion. To assess whether alternate dosing regimens might provide a better reduction in lysosomal storage, a 26-week, randomized, open-label, multinational dose-optimization trial was conducted...
Long-term efficacy and safety of laronidase in the treatment of mucopolysaccharidosis I. [2009.01]
OBJECTIVE: Our goal was to evaluate the long-term safety and efficacy of recombinant human alpha-l-iduronidase (laronidase) in patients with mucopolysaccharidosis I... CONCLUSIONS: This trial demonstrates the long-term clinical benefit and safety of laronidase in attenuated patients with mucopolysaccharidosis I and highlights the magnitude and chronology of treatment effects. Prompt diagnosis and early treatment will maximize treatment outcomes.
Laronidase for treating mucopolysaccharidosis type I. [2007.09.30]
Mucopolysaccharidoses are a group of inherited metabolic diseases caused by the absence or deficiency of the lysosomal enzymes that are needed for breaking down glycosaminoglycans (GAGs).Laronidase seems to be a promising agent for treating mucopolysaccharidosis type I, as shown by the reduction in the urinary excretion of GAGs and the associated improvements in vital capacity and in the performance of defined physical tasks.
Enzyme replacement therapy for mucopolysaccharidosis I: a randomized, double-blinded, placebo-controlled, multinational study of recombinant human alpha-L-iduronidase (laronidase). [2004.05]
OBJECTIVE: To confirm the efficacy and safety of recombinant human alpha-L-iduronidase (laronidase) in patients with mucopolysaccharidosis I (MPS I)... CONCLUSIONS: In patients with MPS I, laronidase significantly improves respiratory function and physical capacity, reduces glycosaminoglycan storage, and has a favorable safety profile.
Use of enzyme replacement therapy (Laronidase) before hematopoietic stem cell transplantation for mucopolysaccharidosis I: experience in 18 patients. [2009.01]
We describe the use of enzyme replacement therapy in conjunction with hematopoietic stem cell transplantation in 18 consecutive patients with severe mucopolysaccharidosis type I. The survival and engraftment rate was 89% overall and 93% for the 15 patients who received full-intensity conditioning..
Clinical Trials Related to Aldurazyme (Laronidase)
A Dose-Optimization Study of Aldurazyme® (Laronidase) in Patients With Mucopolysaccharidosis I (MPS I) Disease [Completed]
The main purpose of this study is to evaluate differences in the pharmacodynamic response of
4 Aldurazyme® (laronidase) dose regimens in patients with Mucopolysaccharidosis I (MPS I)
disease through measuring urinary GAG levels, liver volume, and functional tests (6-minute
walk test).
Phase 3 Extension Study of the Safety and Efficacy of Aldurazyme® (Laronidase) in Mucopolysaccharidosis I (MPS I) Patients [Completed]
This study is being conducted to collect additional long-term efficacy and safety data of
Aldurazyme® (Laronidase) patients with MPS I disease. Patients who were previously enrolled
in the phase 3 double-blind study will be enrolled in this study.
A Study Evaluating the Safety and Pharmacokinetics of Aldurazyme® (Laronidase) in MPS I Patients Less Than 5 Years Old [Completed]
The main objectives of this study are to evaluate the safety and pharmacokinetics (PK) of
enzyme replacement therapy with recombinant human alpha-L-iduronidase [Aldurazyme®
(laronidase)] in mucopolysaccharidosis I (MPS I) patients less than 5 years old. Efficacy
measurements will also be evaluated in this study.
Study of Aldurazyme® Replacement Therapy in Patients With Mucopolysaccharidosis I (MPS I) Disease [Completed]
This is a multi-center, open label, study conducted to evaluate the safety of laronidase
administered by intravenous drip infusion in patients with MPS I disease.
Following baseline evaluation, patients will receive weekly infusions of JC0498 at an
intravenous dose of 100 units/kg. Patient safety will be monitored continuously throughout
the trial. In addition, the effects of JC0498 treatment in this patient population will be
assessed by periodically evaluating aspects of MPS I disease in patients at scheduled
intervals over the duration of the trial.　
Since patients may be eligible for the trial if they have received JC0498, a portion of the
data may be captured retrospectively and recorded onto the case report forms (CRFs).
This study represents the first good clinical practice (GCP) effort to characterize MPS I in
the Japanese population and evaluate the effects of JC0498 on disease manifestations.
A Study Investigating the Relationship Between the Development of Laronidase Antibody and Urinary GAG Levels in Aldurazyme® Treated Patients [Recruiting]
The purpose of this study is to determine whether the development of antibodies to
Aldurazyme® (laronidase) in patients with MPS I receiving Aldurazyme® (laronidase) impairs
the clearance of GAG substrate.
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