Lysosomal acid lipase Inhibitors
1 drugsAbout Lysosomal acid lipase
Lysosomal acid lipase (LAL) is an enzyme within lysosomes that breaks down cholesteryl esters and triglycerides. LAL dysfunction leads to lipid accumulation in tissues and organs, causing various health problems by disrupting cellular homeostasis.
LAL is a therapeutic target because its deficiency causes significant health issues. Currently, there is no genetic evidence available linking LAL to specific diseases, but its role in lipid metabolism makes it a target.
KANUMA is the only FDA-approved drug targeting LAL, developed by ALEXION PHARM. It is a biologic drug addressing a disease area categorized as 'Other'. The first approval was in 2015.
Top Lysosomal acid lipase Drugs
ALEXION PHARM is the only company with an approved drug targeting LAL.
The lack of competition presents a market opportunity, but also highlights potential entry barriers.
Lysosomal acid lipase Drug Modality Landscape
Modalities
Routes of Administration
Only one approved drug targets Lysosomal acid lipase, using enzyme modality.
Explore small molecule or gene therapy approaches to diversify therapeutic options.
Lysosomal acid lipase Clinical Trials 6 trials
Phase 1-4 trials started in 2008 or later that test an FDA-approved drug acting on Lysosomal acid lipase. Source: ClinicalTrials.gov. How we count
Completion by Phase
| Phase | Total | Completed | Stopped | Active | Completion |
|---|---|---|---|---|---|
| Phase 1 | 1 | 1 | 0 | 0 | 100% |
| Phase 2 | 4 | 3 | 1 | 0 | 75% |
| Phase 3 | 1 | 1 | 0 | 0 | 100% |
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Lysosomal acid lipase Drug Approval Timeline (2015 - 2015)
The first and only drug, KANUMA, was approved in 2015.
The approval timeline indicates a nascent market with potential for further drug development.
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Deep insights for drug target analysis
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: 1 (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 Lysosomal acid lipase. A trial counts if an approved drug acting on Lysosomal acid lipase 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.