HAO1 Inhibitors
1 drugsAbout HAO1
Hydroxyacid Oxidase 1 (HAO1) is an enzyme currently being explored as a drug target. Research aims to fully elucidate its role in biological processes.
Currently, there is no genetic evidence directly linking HAO1 to specific diseases. However, the existence of the FDA-approved drug OXLUMO suggests that modulating HAO1 activity can have clinically meaningful effects.
HAO1 is targeted by one FDA-approved drug, OXLUMO, which was first approved in 2020. The drug is an siRNA therapy developed by ALNYLAM PHARMS INC for one indication classified as 'Other'.
Top HAO1 Drugs
The competitive landscape is dominated by ALNYLAM PHARMS INC, the sole company with an approved HAO1-targeting drug.
High market concentration suggests high entry barriers, requiring differentiated strategies for new entrants.
HAO1 Drug Modality Landscape
Modalities
Routes of Administration
Only one approved drug targets HAO1, using sirna modality.
The exclusive use of siRNA presents an opportunity to explore alternative modalities like small molecules or antibodies.
HAO1 Clinical Trials 7 trials
Phase 1-4 trials started in 2008 or later that test an FDA-approved drug acting on HAO1. Source: ClinicalTrials.gov. How we count
Completion by Phase
| Phase | Total | Completed | Stopped | Active | Completion |
|---|---|---|---|---|---|
| Phase 1 | 1 | 1 | 0 | 0 | 100% |
| Phase 2 | 3 | 1 | 1 | 1 | 50% |
| Phase 3 | 3 | 3 | 0 | 0 | 100% |
Top Sponsors
By Modality
Top Conditions
Top Drugs
HAO1 Drug Approval Timeline (2020 - 2020)
OXLUMO was the first and most recent drug approved in 2020, representing a short approval history.
The recent approval suggests potential for further drug development, but also indicates early stage market maturity.
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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: 2 (1 company)
- • 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 HAO1. A trial counts if an approved drug acting on HAO1 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.