Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
GA-GCB · 2 trials · 1 indication
Safety was assessed throughout the study by assessments including adverse events, concomitant medication use, and vital signs. Additional safety assessments, including 12-lead ECGs, physical examinations, clinical laboratory tests and determination of the presence of anti-velaglucerase alfa antibodies. Refer to Adverse event section for further details.
Overall Summary of Treatment-emergent Adverse Events-Safety Population
| Arm | Type | Description |
|---|---|---|
| GA-GCB (velaglucerase alfa) | EXPERIMENTAL | 15-60 U/kg, every other week via intravenous infusion |
| GA-GCB | EXPERIMENTAL | 15-60 U/kg every other week via intravenous infusion |
| Name | Type | Description |
|---|---|---|
| GA-GCB (velaglucerase alfa) | BIOLOGICAL | 15-60 U/kg, every other week via intravenous infusion |
| GA-GCB | DRUG | 15-60 U/kg every other week via intravenous infusion |
Inclusion Criteria: Includes: * The participant has a documented diagnosis of type 1 Gaucher disease, as determined by deficient glucocerebrosidase (GCB) activity relative to normal as measured in leukocytes or by genotype analysis and the participant/legal guardian is willing and able to provide ...
GA-GCB is an investigational small molecule being developed for Gaucher disease, a rare inherited metabolic disorder. It is studied as an enzyme replacement therapy for patients with Type 1 Gaucher disease, including those previously treated with imiglucerase. The drug is in clinical development and has not been approved by regulatory authorities.
GA-GCB is being developed by Takeda Pharmaceutical Company Limited, which trades under the ticker TAK. Takeda is conducting clinical trials to evaluate the safety and efficacy of GA-GCB in patients with Type 1 Gaucher disease. The drug is currently in Phase 2 clinical development.
GA-GCB is in Phase 2 clinical development. It has completed two clinical trials, including a Phase 1 open-label extension study and a Phase 2 study in patients with Type 1 Gaucher disease. The drug is investigational and has not received FDA approval.
GA-GCB has completed two clinical trials. NCT00391625 was a Phase 1 open-label extension study evaluating long-term safety in patients with Type 1 Gaucher disease receiving DRX008A, with 10 participants. NCT00478647 was a Phase 2 study of GA-GCB enzyme replacement therapy in patients previously treated with imiglucerase, with 40 participants.
GA-GCB is an investigational small molecule enzyme replacement therapy being developed for Gaucher disease, a rare genetic disorder. It is designed to replace the deficient enzyme in patients with Type 1 Gaucher disease. The drug is currently in Phase 2 clinical development by Takeda Pharmaceutical Company and has completed two trials.