ALDURAZYME (laronidase)
ALDURAZYME is indicated for the treatment of Mucopolysaccharidosis I.
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.
Development Insights
Details
- Status
- Prescription
- First Approved
- 2003-04-30
- Patent Cliff
- 2010
- Revenue
- $58M (Q4-2025)
- 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) ] .
ALDURAZYME Competitive Set
ProThree rings of competition based on shared molecular targets and treated indications.
MoA expansion candidates
Same target(s), different indications — where else is this mechanism being explored?
Filters applied: drops same-active-ingredient (505(b)(2) reformulations), route-mismatch (topical vs systemic), and cross-therapeutic-area matches in same-indication rings.
Clinical Trial Registry
6 trials| Trial | Sponsor ID | Phase | Status | Title |
|---|---|---|---|---|
| NCT05134571 | LPS16578 U1111-1260-3947 | Ph 4 | completed | China Post-marketing Surveillance (PMS) Study of Aldurazyme® |
| NCT06406153 | CIN.LAR.AR.III.97 | Ph 3 | completed | Efficacy and Safety of YW17 (Laronidase-CinnaGen) Compared to Aldurazyme® in MPS I Patients |
| NCT00418821 results posted | OBS12874 2007-007003-33, ALID01803 | Ph 4 | terminated | A Study of the Effect of Aldurazyme® (Laronidase) Treatment on Lactation in Female Patients With Mucopolysaccharidosis I (MPS I) and Their Breastfed Infants |
| NCT01173016 results posted | 2009LS090 MT2009-20, 1004M80513 | Ph 1 | completed | Administration of IV Laronidase Post Bone Marrow Transplant in Hurler |
| NCT00741338 results posted | ALID02307 2007-001163-30 | Ph 1, Ph 2 | completed | Immune Tolerance Study With Aldurazyme® (Laronidase) |
| NCT00786968 | MIRC-001-01 | Ph 1 | terminated | Extension Study of Intrathecal Enzyme Replacement Therapy for MPS I |
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
Indications & Usage
FDA Label (PDF)ALDURAZYME is indicated for the treatment of Mucopolysaccharidosis I.
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...
Pro Intelligence Preview
Deep insights for ALDURAZYME
Revenue Insights
- • Q4-2025: $58M
- • Historical trend analysis
Patent Timeline
- • Cliff: 2010
- • Generic/biosimilar risk
Trial Analysis
- • 6 total trials
- • Stage: Declining
Competitive Landscape
- • Competitor tracking
- • Same target/indication analysis
Full approval history • All patents • Revenue trends • Competitor analysis
Data Sources
Data sourced from official FDA and NIH databases. Click links to verify on original sources.
How We Calculate These Metrics
Trial Activity Stage
Measures the current development activity pattern based on trial phases, status, and trends. Important: This measures R&D activity, not commercial lifecycle.
Trial statuses: "Active" means recruiting or ongoing. "Completed" means reached planned endpoint. "Terminated" means stopped early—often due to safety, efficacy, or business reasons.
- Growth: High proportion of early-phase trials (Phase 1/2), active development
- Expansion: Significant Phase 3 activity, approaching or pursuing approvals
- Mature: Substantial Phase 4 post-marketing studies
- Stable: Mixed phase distribution, steady development
- Declining: Low active trial ratio, reduced R&D investment