ALDURAZYME
ALDURAZYME (laronidase) is a hydrolytic lysosomal glycosaminoglycan-specific enzyme indicated for the treatment of adult and pediatric patients with Mucopolysaccharidosis I (MPS I). The therapy is approved for patients with the Hurler and Hurler-Scheie forms of the disease, as well as those with the Scheie form who exhibit moderate to severe symptoms. It is intended to provide exogenous enzyme replacement to address systemic manifestations of the disorder, though its impact on central nervous system symptoms has not been established. The safety and effectiveness of the drug have not been determined for mildly affected patients with the Scheie form.
How ALDURAZYME Works
Mucopolysaccharidosis I is caused by a deficiency of the lysosomal enzyme α-L-iduronidase, which is required to break down glycosaminoglycans (GAG) such as dermatan sulfate and heparan sulfate. ALDURAZYME provides an exogenous version of this enzyme that is taken up into cellular lysosomes, likely through binding with mannose-6-phosphate receptors. Once inside the lysosome, the enzyme catalyzes the hydrolysis of accumulated GAG, thereby increasing their catabolism and reducing cellular and organ dysfunction. However, the enzyme is restricted from entering the central nervous system by the blood-brain barrier, limiting its activity to peripheral tissues.
Details
- Status
- Prescription
- First Approved
- 2003-04-30
- Routes
- N/A
- Dosage Forms
- VIAL
ALDURAZYME Approval History
What ALDURAZYME Treats
1 indicationsALDURAZYME is approved for 1 conditions since its original approval in 2003. These indications span multiple therapeutic areas including oncology, immunology, and more.
- Mucopolysaccharidosis I
ALDURAZYME Boxed Warning
HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS, and ACUTE RESPIRATORY COMPLICATIONS ASSOCIATED WITH ADMINISTRATION WARNING: HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS, and ACUTE RESPIRATORY COMPLICATIONS ASSOCIATED WITH ADMINISTRATION See full prescribing information for complete boxed warning . Appropriate medical monitoring and support measures, including cardiopulmonary resuscitation equipment, should be readily available. If a severe hypersensitivity reaction occurs, discontinue ALDU...
WARNING: HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS, and ACUTE RESPIRATORY COMPLICATIONS ASSOCIATED WITH ADMINISTRATION WARNING: HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS, and ACUTE RESPIRATORY COMPLICATIONS ASSOCIATED WITH ADMINISTRATION See full prescribing information for complete boxed warning . Appropriate medical monitoring and support measures, including cardiopulmonary resuscitation equipment, should be readily available. If a severe hypersensitivity reaction occurs, discontinue ALDURAZYME immediately and initiate appropriate medical treatment. ( 5.1 ) Patients with compromised respiratory function or acute respiratory disease may be at risk of serious acute exacerbation of their respiratory compromise due to infusion reactions and require additional monitoring. Appropriate respiratory support should be available during infusion. ( 5.2 ) Hypersensitivity Reactions Including Anaphylaxis Patients treated with ALDURAZYME have experienced life-threatening hypersensitivity reactions, including anaphylaxis. Appropriate medical monitoring and support measures, including cardiopulmonary resuscitation equipment, should be readily available during ALDURAZYME administration. If a severe hypersensitivity reaction (e.g., anaphylaxis) occurs, discontinue ALDURAZYME immediately and initiate appropriate medical treatment. In patients with severe hypersensitivity reactions, a desensitization procedure to ALDURAZYME may be considered [see Warnings and Precautions (5.1) ] . Acute Respiratory Complications Associated with Administration Patients with compromised respiratory function or acute respiratory disease may be at risk of serious acute exacerbation of their respiratory compromise due to infusion reactions and require additional monitoring [see Warnings and Precautions (5.2) ] .
Active Pipeline
Ongoing clinical trials by development phase
Key Completed Trials
Completed studies with published results, ranked by significance
Trial Timeline
Full development history with FDA approval milestones
Understanding FDA Approval Types
| Count | Type | What it means |
|---|---|---|
| - | ORIG | Original approval - drug first enters market |
| - | SUPPL - Efficacy | New indication (new disease/condition approved) |
| - | SUPPL - Labeling | Label text changes (warnings, dosing updates) |
| - | SUPPL - Manufacturing | Production changes (new facility) |
| - | SUPPL - Chemistry | Formulation changes (new dosage strength) |
Green lines in the timeline show ORIG and Efficacy approvals - the clinically meaningful milestones.
ALDURAZYME FDA Label Details
ProIndications & Usage
FDA Label (PDF)ALDURAZYME ® is indicated for the treatment of: adult and pediatric patients with Hurler and Hurler-Scheie forms of Mucopolysaccharidosis I (MPS I) and patients with the Scheie form of MPS I who have moderate to severe symptoms. ALDURAZYME is a hydrolytic lysosomal glycosaminoglycan (GAG)-specific enzyme indicated for the treatment of adult and pediatric patients with Hurler and Hurler-Scheie forms of Mucopolysaccharidosis I (MPS I) and for the treatment of patients with the Scheie form of MPS I who have moderate to severe symptoms. Limitations of Use: The risks and benefits of treating mildly...
WARNING: HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS, and ACUTE RESPIRATORY COMPLICATIONS ASSOCIATED WITH ADMINISTRATION WARNING: HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS, and ACUTE RESPIRATORY COMPLICATIONS ASSOCIATED WITH ADMINISTRATION See full prescribing information for complete bo...
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Data Sources
Data sourced from official FDA and NIH databases. Click links to verify on original sources.