Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Afegostat · 3 trials · 3 indications
A TEAE was defined as any adverse event (AE) with start date on or after administration of study drug or pre-existing conditions that worsened on or after the start of the first study drug administration (on Day 1). A severe AE defined as an AE that was incapacitating and required medical intervention. The number of participants who experienced 1 or more severe TEAEs after dosing on Day 1 through the end of follow-up is presented. A summary of serious and all other non-serious AEs regardless of causality is located in the Reported Adverse Events module.
| Arm | Type | Description |
|---|---|---|
| Afegostat Tartrate Treatment Regimen 1 | EXPERIMENTAL | Afegostat tartrate was administered orally at a dose of 225 mg QD for 3 or 7 consecutive days followed by no study medication for 4 or 7 consecutive days (consecutive 3-days-on/4-days-off or 7-days-on/7-days-off, respectively). Amendment 2 added a MWF 3-days-on/4-days-off regimen. After Amendment 2 was implemented, all participants were assigned to one of the two 3-days-on/4-days-off regimens. Amendment 4 removed the consecutive 3-days-on/4-days-off regimen, and all participants were assigned to the MWF 3-days-on/4-days-off regimen. Participants were to receive afegostat tartrate for 30 months and be followed for 6 months after EOT. |
| Afegostat Tartrate Treatment Regimen 2 | EXPERIMENTAL | Afegostat tartrate was administered orally at a dose of 225 mg QD for 7 consecutive days, followed by no study medication for 7 consecutive days. This 7-days-on/7-days-off treatment regimen was followed for 24 weeks. |
| Afegostat tartrate 25 milligrams (mg) once per day | EXPERIMENTAL | Afegostat tartrate was administered orally during the 4-week treatment period. |
| Afegostat tartrate 150 mg once per day | EXPERIMENTAL | Afegostat tartrate was administered orally once per day during the 4-week treatment period. |
| Afegostat tartrate 150 mg once every four days | EXPERIMENTAL | Afegostat tartrate was administered orally once every 4 days during the 4-week treatment period. |
| Afegostat tartrate 150 mg once every seven days | EXPERIMENTAL | Afegostat tartrate was administered orally once every 7 days during the 4-week treatment period. |
| Name | Type | Description |
|---|---|---|
| afegostat tartrate | DRUG | - |
Inclusion Criteria: * Male or female participants, 18 years of age or older * Completed study GAU-CL-202 with no significant protocol violations or safety concerns * Clinically stable * Had not received enzyme replacement therapy (ERT) or substrate reduction therapy (SRT) in the past 12 months and ...
Afegostat is an investigational small molecule being developed for Gaucher disease, including Gaucher disease type 1. It is a pharmacological chaperone intended to treat this rare lysosomal storage disorder. The drug is currently in Phase 2 clinical development and has not been approved by regulatory authorities.
Afegostat is being developed by Amicus Therapeutics, Inc., a biopharmaceutical company traded on NASDAQ under the ticker symbol FOLD. The company is focused on developing treatments for rare and orphan diseases, with Afegostat being one of its investigational small molecule candidates for Gaucher disease.
Afegostat is in Phase 2 clinical development. It is an investigational drug and has not received regulatory approval. The clinical program includes completed Phase 2 studies evaluating the drug in patients with Gaucher disease type 1, including both treatment-naive patients and those currently receiving enzyme replacement therapy.
Afegostat has been studied in three completed Phase 2 clinical trials. NCT00433147 evaluated the drug in 30 adult patients with type 1 Gaucher disease receiving enzyme replacement therapy in the United States. NCT00446550 studied 19 treatment-naive patients across the US, Israel, South Africa, and the UK. NCT00813865 was a long-term extension study enrolling 8 patients.
Afegostat is a small molecule pharmacological chaperone designed to bind to and stabilize the defective enzyme glucocerebrosidase in Gaucher disease. By enhancing the enzyme's proper folding and trafficking, it aims to restore enzyme activity and reduce the accumulation of substrate that causes disease symptoms. This mechanism targets the underlying enzyme deficiency in the condition.