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Afegostat

Alternative Names: afegostat, at-2101, at2101, at 2101, plicera
Latest Update: 2023-12-18
Latest Update Note: News Article

Product Description

Afegostat tartrate (also known as AT2101 or isofagomine tartrate) is designed to act as a pharmacological chaperone by selectively binding to misfolded β-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

Map of Global Clinical Trials for Afegostat

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

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