GBA1
Lysosomal acid glucosylceramidase
As of the edition of 20 October 2026, GBA1 (lysosomal acid glucosylceramidase, UniProt P04062) has 2 drugs tested at it and 6 companies on the list, 1 on route A and 0 on route B.
Sources: UniProt, ClinicalTrials.gov, ChEMBL, FDA Orange Book and Purple Book, Pharos.
The protein
- UniProt
- P04062
- How well studied
- Drug target: an approved drug acts on it
- Where it sits in the cell
- At a membrane
- Solved structures
- 58
- Disease areas
- Nervous system; Nutrition and metabolism; Inherited and neonatal; Lungs and airways; Infections; Ear, nose and throat; Mental health; Mouth and teeth
- Registered trials of its drugs
- 31
Drugs at this target
| Status | Count |
|---|---|
| Marketed | 1 |
| Went quiet | 1 |
| Total | 2 |
| Drug | Status | Furthest phase | US protection |
|---|---|---|---|
| Ambroxol | Marketed | Phase 4 | none listed |
| Afegostat | Went quiet | Phase 2 | none listed |
2 of 2 drugs, the most tested first.
Companies at this target
| Where the company stands | Companies |
|---|---|
| Route A | 1 |
| Undetermined | 5 |
| Total on the list | 6 |
| Company | Route |
|---|---|
| Amicus Therapeutics | Route A |
The other 5 companies at GBA1
In the product6 companies in all; 5 are not named here, and no score is.
See the 6 companies at GBA1, ranked, with the facts behind eachRequest access. Nothing is shown until you do.