Glucocerebrosidase Inhibitors
2 drugsAbout Glucocerebrosidase
Glucocerebrosidase (GCase) is a lysosomal enzyme responsible for breaking down glucocerebroside. Deficiency leads to glucocerebroside accumulation, primarily in macrophages, causing cellular dysfunction.
GCase is a therapeutic target for rare diseases caused by its deficiency. There is currently no genetic evidence data available for this target.
Two FDA-approved drugs, ELELYSO and CEREZYME, target GCase for rare diseases. Both drugs are biologics (other) developed by Pfizer and Sanofi, respectively.
Top Glucocerebrosidase Drugs
The competitive landscape is concentrated with Pfizer and Sanofi holding all approved drugs.
High market concentration suggests significant barriers to entry for new competitors.
Glucocerebrosidase Drug Modality Landscape
Modalities
Routes of Administration
Glucocerebrosidase requires biologic approaches (enzyme), likely due to its structure or location.
The lack of small molecule or antibody drugs suggests a whitespace opportunity for novel modalities.
Glucocerebrosidase Clinical Trials 15 trials
Phase 1-4 trials started in 2008 or later that test an FDA-approved drug acting on Glucocerebrosidase. Source: ClinicalTrials.gov. How we count
Completion by Phase
| Phase | Total | Completed | Stopped | Active | Completion |
|---|---|---|---|---|---|
| Phase 1 | 1 | 1 | 0 | 0 | 100% |
| Phase 2 | 1 | 0 | 0 | 1 | - |
| Phase 3 | 9 | 7 | 1 | 1 | 88% |
| Phase 4 | 4 | 3 | 1 | 0 | 75% |
Top Sponsors
By Modality
Top Conditions
Glucocerebrosidase Drug Approval Timeline (1994 - 2012)
The first drug was approved in 1994, and the most recent in 2012, spanning 19 years.
The slow approval rate and long span suggest a saturated market with limited recent innovation.
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Competitive Landscape
- • 2 companies competing
- • Market share by company
Full Drug Portfolio
- • All 2 approved drugs
- • Approval dates & indications
Genetic Validation
- • Full genetic evidence table
- • Effect sizes & directions
Approval Timeline
- • Full 2-drug timeline
- • First-of-modality markers
Clinical Trials Analysis
- • Sponsors: 6 (5 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 Glucocerebrosidase. A trial counts if an approved drug acting on Glucocerebrosidase 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.