Product Description
Afegostat tartrate (also known as AT2101 or isofagomine tartrate) is designed to act as a pharmacological chaperone by selectively binding to misfolded beta-glucocerebrosidase (GCase) and helping it fold correctly, intended to restore GCase activity. (Sourced from: https://clinicaltrials.gov/ct2/show/NCT00433147)
Mechanisms of Action: GCase Binder
Novel Mechanism: Yes
Modality: Small Molecule
Route of Administration: Oral
FDA Designation: None *
Approval Status: Not Approved
Approved Countries: None
Approved Indications: None
Known Adverse Events: None
Company: Amicus
Company Location: PHILADELPHIA PA 19104
Company CEO: Bradley L. Campbell
Additonal Commercial Interests: None
Clinical Description
Countries in Clinic:
Active Clinical Trial Count:
Highest Development Phases
Phase 2: Gaucher Disease
Trial |
Phase |
Trial Status |
Disease |
Primary Completion Date |
Probability of Success |
|---|---|---|---|---|---|
| 2008-007158-36 | P2 |
Terminated |
Gaucher Disease |
2013-03-25 |
|
| GAU-CL-202X | P2 |
Completed |
Gaucher Disease |
2012-05-01 |
|
| 2007-003359-35 | P2 |
Completed |
Gaucher Disease |
2009-11-16 |
|
| GAU-CL-202 | P2 |
Completed |
Gaucher Disease |
2009-08-20 |