alpha-L-iduronidase Inhibitors
1 drugsAbout alpha-L-iduronidase
Alpha-L-iduronidase is a lysosomal enzyme that breaks down glycosaminoglycans (GAGs). Deficiency leads to GAG accumulation and lysosomal storage disorders.
Alpha-L-iduronidase is a therapeutic target for lysosomal storage disorders, but there is currently no genetic evidence data available for this target.
ALDURAZYME, a biologic drug, was approved in 2003 and is the only approved drug targeting alpha-L-iduronidase. BIOMARIN is the only company with an approved drug.
Top alpha-L-iduronidase Drugs
BIOMARIN is the only company with an approved drug targeting this enzyme.
The market is highly concentrated, indicating high barriers to entry or limited commercial opportunity.
alpha-L-iduronidase Drug Modality Landscape
Modalities
Routes of Administration
Only one approved drug targets alpha-L-iduronidase, using enzyme modality.
The lack of modality diversity suggests an opportunity for novel therapeutic approaches.
alpha-L-iduronidase Clinical Trials 8 trials
Phase 1-4 trials started in 2008 or later that test an FDA-approved drug acting on alpha-L-iduronidase. Source: ClinicalTrials.gov. How we count
Completion by Phase
| Phase | Total | Completed | Stopped | Active | Completion |
|---|---|---|---|---|---|
| Phase 1 | 5 | 3 | 1 | 1 | 75% |
| Phase 3 | 1 | 1 | 0 | 0 | 100% |
| Phase 4 | 2 | 1 | 1 | 0 | 50% |
Top Sponsors
By Modality
Top Conditions
Top Drugs
alpha-L-iduronidase Drug Approval Timeline (2003 - 2003)
ALDURAZYME was approved in 2003 and remains the only approved drug.
The absence of recent approvals suggests market saturation or challenges in developing new therapies.
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Competitive Landscape
- • 1 companies competing
- • Market share by company
Full Drug Portfolio
- • All 1 approved drugs
- • Approval dates & indications
Genetic Validation
- • Full genetic evidence table
- • Effect sizes & directions
Approval Timeline
- • Full 1-drug timeline
- • First-of-modality markers
Clinical Trials Analysis
- • Sponsors: 5 (2 companies)
- • Completion rates by condition
Full summary • All drugs • Genetic evidence • Trials • Timeline
How We Count Trials
- Trials: phase 1-4 interventional trials on ClinicalTrials.gov that started in 2008 or later and test an FDA-approved drug acting on alpha-L-iduronidase. A trial counts if an approved drug acting on alpha-L-iduronidase is in any arm, including as a comparator, and a trial that tests two such drugs counts once. Trials that test only unapproved drugs are not counted. A trial listed as phase 1/2 counts as phase 1, and phase 2/3 as phase 2.
- Active: recruiting, not yet recruiting, enrolling by invitation, or active and no longer recruiting.
- Completion rate: of the trials that have ended, the share marked Completed rather than Terminated or Withdrawn. Completed means the trial ended as planned; it does not mean the drug worked. Suspended trials are not counted as ended.
- Stopped: terminated or withdrawn. Trials stop for many reasons, including slow enrolment and business decisions, not only safety or efficacy.
- Sponsors: the lead sponsor of each trial. Companies are the sponsors ClinicalTrials.gov classes as industry; the rest are universities, hospitals, cooperative groups, government bodies and individuals.