Afegostat
As of the edition of 20 October 2026, Afegostat is a small molecule: went quiet in 2012; acts on GBA1.
Sources: FDA Orange Book and Purple Book, other approval registers, ClinicalTrials.gov, ChEMBL.
The drug
- Status
- Went quiet, 2012
- Kind
- Small molecule
- Registered trials
- 4, 1 stopped early
- Disease areas
- Inherited and neonatal; Nervous system; Nutrition and metabolism
US protection
No US approval on record.
With no US approval on record there are no listed US patents or exclusivities.
This is the US only.
Companies that need a new molecule because of this drug
1 company on route A because of this drug.
Companies on route A for this drug
In the product1 company is on route A because of Afegostat.
See the 1 company on route A for this drug, with the facts behind eachRequest access. Nothing is shown until you do.
Target
| Gene | Protein | UniProt |
|---|---|---|
| GBA1 | Lysosomal acid glucosylceramidase | P04062 |
Registered trials
| Phase | Trials | Completed | Active | Stopped early |
|---|---|---|---|---|
| Phase 1 | 1 | 0 | 0 | 1 |
| Phase 2 | 3 | 3 | 0 | 0 |
| All trials | 4 |
Who ran its trials
| Company | Role | Years |
|---|---|---|
| Amicus Therapeutics | Lead sponsor of trials | 2007 to now |