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: *
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
Additional Commercial Interests: None
Clinical Description
Countries in Clinic:
Active Clinical Trial Count:
Recent & Upcoming Milestones
Highest Development Phases
Phase 2: Gaucher Disease
Trial |
Phase |
Trial Status |
Disease |
Primary Completion Date |
Probability of Success |
Latest Trial Update Date |
Data Updated |
|---|---|---|---|---|---|---|---|
| GAU-CL-104 | P1 |
Terminated |
Gaucher Disease |
2009-07-01 |
2019-03-18 |
Treatments |
|
| 2008-007158-36 | P2 |
Terminated |
Gaucher Disease |
2013-03-25 |
2025-06-13 |
Treatments |
|
| GAU-CL-202X | P2 |
Completed |
Gaucher Disease |
2012-05-01 |
2019-03-18 |
Treatments |
|
| 2007-003359-35 | P2 |
Completed |
Gaucher Disease |
2009-11-16 |
2022-03-12 |
Treatments |
|
| GAU-CL-202 | P2 |
Completed |
Gaucher Disease |
2009-08-20 |
2019-03-21 |
Treatments |
|
| GAU-CL-201 | P2 |
Completed |
Gaucher Disease |
2008-02-19 |
2019-03-21 |
Treatments |
Recent News Events
Date |
Type |
Title |
|---|---|---|
|
06/18/2025 |
News Article |
NEW ORLEANS' HISTORIC THEATRE AUCTION JULY 17-23 |
|
04/21/2025 |
News Article |
Rural King Opens New Kingsport Store, Bringing Farm and Home Essentials to the Region |
|
12/04/2024 |
News Article |
Sunstone Credit Powers Solar Installation Project at Baltimore's 2101 E. Biddle Street |
|
10/18/2024 |
News Article |
REI Co-op announces lineup of new store openings in 2025 and an early peek at 2026 |
|
04/01/2024 |
PubMed |
Expression and metabolism profiles of CVT associated with inflammatory responses and oxygen carrier ability in the brain. |
|
02/01/2021 |
PubMed |
Re-recognizing bromhexine hydrochloride: pharmaceutical properties and its possible role in treating pediatric COVID-19. |
|
05/09/2020 |
PubMed |
Synthesis and Therapeutic Applications of Iminosugars in Cystic Fibrosis. |