alpha-galactosidase A Inhibitors
3 drugsAbout alpha-galactosidase A
Alpha-galactosidase A (α-Gal A) is a lysosomal hydrolase enzyme that breaks down globotriaosylceramide (Gb3). Deficiency leads to Gb3 accumulation in cells, causing dysfunction and disease.
α-Gal A is a therapeutic target for diseases characterized by Gb3 accumulation; however, there is currently no genetic evidence directly linking it to disease.
Two FDA-approved biologics, ELFABRIO and FABRAZYME, target α-Gal A for rare diseases, developed by CHIESI FARMACEUTICI SPA and Sanofi, respectively.
Top alpha-galactosidase A Drugs
The competitive landscape is concentrated, with only two companies holding approved drugs.
High market concentration suggests significant barriers to entry for new competitors.
alpha-galactosidase A Drug Modality Landscape
Modalities
Routes of Administration
alpha-galactosidase A is druggable by both biologics (2) and small molecules (1), indicating broad therapeutic accessibility.
Consider exploring non-biologic modalities to differentiate from existing therapies.
📈 Modality Evolution
Enzymes pioneered alpha-galactosidase A targeting (2003), with small molecules entering more recently (2018).
alpha-galactosidase A Clinical Trials 19 trials
Phase 1-4 trials started in 2008 or later that test an FDA-approved drug acting on alpha-galactosidase A. Source: ClinicalTrials.gov. How we count
Completion by Phase
| Phase | Total | Completed | Stopped | Active | Completion |
|---|---|---|---|---|---|
| Phase 1 | 2 | 2 | 0 | 0 | 100% |
| Phase 2 | 1 | 1 | 0 | 0 | 100% |
| Phase 3 | 12 | 9 | 1 | 2 | 90% |
| Phase 4 | 4 | 3 | 1 | 0 | 75% |
Top Sponsors
By Modality
alpha-galactosidase A Drug Approval Timeline (2003 - 2023)
The first drug was approved in 2003, with the most recent approval in 2023, spanning 21 years.
The approval timeline indicates a slow but steady progression, suggesting potential for further innovation.
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Competitive Landscape
- • 3 companies competing
- • Market share by company
Full Drug Portfolio
- • All 3 approved drugs
- • Approval dates & indications
Genetic Validation
- • Full genetic evidence table
- • Effect sizes & directions
Approval Timeline
- • Full 3-drug timeline
- • First-of-modality markers
Clinical Trials Analysis
- • Sponsors: 7 (7 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-galactosidase A. A trial counts if an approved drug acting on alpha-galactosidase A 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.