Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
GR181413A/AT1001 · 1 trial · 1 indication
AUC, Cmax, tmax, Tlast , t1/2, CL/F, Vz/F
Ae, CLr, %Fx
| Arm | Type | Description |
|---|---|---|
| 50 mg | EXPERIMENTAL | GR181413A/AT1001 |
| 150 mg | EXPERIMENTAL | GR181413A/AT1001 |
| 450 mg | EXPERIMENTAL | GR181413A/AT1001 |
| Placebo | PLACEBO_COMPARATOR | placebo |
| Name | Type | Description |
|---|---|---|
| GR181413A/AT1001 solution | DRUG | Powder for reconstitution |
| GR181413A/AT1001 capsule | DRUG | Size 2, hard gelatin capsule, white opaque / blue opaque |
| Potable water | OTHER | Matched, Size 2, hard gelatin capsule, white opaque/blue opaque |
| Placebo capsule | DRUG | Solution matched |
Inclusion Criteria: 1. Healthy as determined by a responsible and experienced physician, based on a medical evaluation including medical history, physical examination, laboratory tests and cardiac monitoring. A subject with a clinical abnormality or laboratory parameters outside the reference range...
GR181413A/AT1001 is an investigational small molecule developed by Amicus Therapeutics, Inc. (ticker: FOLD) for Fabry disease, a rare genetic disorder. It is in Phase 1 clinical development and has been studied in a single completed trial. The drug is also known as GR181413A/AT1001 solution.
GR181413A/AT1001 is being developed for the treatment of Fabry disease, a rare lysosomal storage disorder. It has not been approved for any indication and remains investigational. The only clinical trial conducted to date evaluated the drug in healthy Japanese subjects, not in patients with Fabry disease.
GR181413A/AT1001 is developed by Amicus Therapeutics, Inc., which trades on the Nasdaq under the ticker FOLD. Amicus is a biotechnology company focused on rare diseases. The company sponsored the Phase 1 trial of GR181413A/AT1001.
GR181413A/AT1001 is in Phase 1 clinical development. It has completed one Phase 1 trial and has no active trials. The drug is investigational and has not been approved by the FDA or any regulatory agency for Fabry disease or any other indication.
GR181413A/AT1001 has been studied in one completed Phase 1 trial, NCT01853852. This randomized, single-blind, placebo-controlled, four-period crossover, dose-escalation study evaluated the safety and pharmacokinetics of single oral doses in 14 healthy Japanese subjects in Australia. The trial is completed and no active trials are ongoing.