Glucocerebroside Inhibitors
1 drugsAbout Glucocerebroside
Glucocerebroside (GlcCer) is a lipid molecule crucial for cellular membranes and biological processes. Aberrant accumulation is a hallmark of lysosomal storage disorders.
Targeting Glucocerebroside is a therapeutic strategy for rare diseases, particularly Gaucher disease, by modulating its levels in the glycosphingolipid metabolic pathway. There is currently no genetic evidence data available for this target.
Glucocerebroside is targeted by one FDA-approved drug, VPRIV (Takeda), a biologic (other) used in rare disease. VPRIV was first approved in 2010.
Top Glucocerebroside Drugs
Takeda is the only company with an approved drug targeting Glucocerebroside.
High market concentration indicates potential entry barriers but also whitespace opportunity.
Glucocerebroside Drug Modality Landscape
Modalities
Routes of Administration
Only one approved drug targets Glucocerebroside, using enzyme modality.
Explore alternative modalities like small molecules to diversify the therapeutic approach.
Glucocerebroside Clinical Trials 10 trials
Phase 1-4 trials started in 2008 or later that test an FDA-approved drug acting on Glucocerebroside. Source: ClinicalTrials.gov. How we count
Completion by Phase
| Phase | Total | Completed | Stopped | Active | Completion |
|---|---|---|---|---|---|
| Phase 1 | 1 | 1 | 0 | 0 | 100% |
| Phase 3 | 5 | 5 | 0 | 0 | 100% |
| Phase 4 | 4 | 3 | 1 | 0 | 75% |
Top Sponsors
By Modality
Top Conditions
Top Drugs
Glucocerebroside Drug Approval Timeline (2010 - 2010)
The first and only drug, VPRIV, was approved in 2010.
The approval timeline suggests market saturation and limited innovation in this area.
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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: 4 (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 Glucocerebroside. A trial counts if an approved drug acting on Glucocerebroside 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.