Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
VPRIV · 2 trials · 1 indication
Safety was evaluated by an analysis of adverse events (AEs), concomitant medication use, clinical laboratory tests, vital signs during the infusion of study drug, physical examination, and the development of anti-velaglucerase alfa. No formal comparisons or statistical tests were applied for the safety analyses, including for differences between the groups.
Efficacy endpoint
| Arm | Type | Description |
|---|---|---|
| VPRIV 60 U/kg(VPRIV Parent Study 45 or 60 U/kg- TKT032,GCB039) | EXPERIMENTAL | This arm is the Overall velaglucerase alfa (VPRIV) 60 U/kg and includes patients from the following groups: VPRIV 45 U/kg or 60 U/kg, IV, EOW for 51 weeks in parent study TKT032 (NCT00430625) and switched to 60 U/kg in HGT-GCB-044 to maintain blindness or 60 U/kg, IV, EOW for 39 weeks in parent study HGT-GCB-039 (NCT00553631) |
| VPRIV 60 U/kg (Parent study-imiglucerase(60 U/kg) HGT-GCB-039) | EXPERIMENTAL | imiglucerase 60 U/kg, IV, EOW for 39 weeks in parent study HGT-GCB-039 (NCT00553631)and switched 60 U/kg VPRIV in HGT-GCB-044 |
| VPRIV 15-60 U/kg (Parent study VPRIV (15-60 U/kg) TKT034) | EXPERIMENTAL | VPRIV 15- 60 U/kg, IV, EOW for 51 weeks in parent study TKT034 (NCT00478647) and continued in HGT-GCB-044 at the same dose as prescribed in TKT034 |
| VPRIV®-45 U/kg, IV, every other week | EXPERIMENTAL | VPRIV® (velaglucerase alfa, Gene Activated® human glucocerebrosidase, GA-GCB) |
| VPRIV®-60 U/kg, IV, every other week | EXPERIMENTAL | VPRIV® (velaglucerase alfa, Gene Activated® human glucocerebrosidase,GA-GCB) |
| Name | Type | Description |
|---|---|---|
| VPRIV® | BIOLOGICAL | Intravenous infusion, every other week (EOW) |
| VPRIV ®, | BIOLOGICAL | Intravenous (IV) infusion, every other week via intravenous infusion for 12 months |
Inclusion Criteria: 1. The patient has completed study TKT032 or TKT034, or study HGT-GCB-039. 2. Female patients of child-bearing potential must agree to use a medically acceptable method of contraception at all times during the study and must have negative results to a pregnancy test performed at...
VPRIV is used for the treatment of Gaucher Disease, Type 1. It is an enzyme replacement therapy being developed by Takeda Pharmaceutical Company Limited. VPRIV is currently in Phase 3 clinical development and is considered investigational, meaning it has not yet been approved by regulatory authorities.
VPRIV is a monoclonal antibody that targets glucocerebrosidase, an enzyme deficient in patients with Gaucher Disease, Type 1. By providing this enzyme, VPRIV aims to address the underlying metabolic defect. The drug is administered as an enzyme replacement therapy to help manage the condition.
VPRIV is developed by Takeda Pharmaceutical Company Limited, which trades under the ticker symbol TAK. The company is conducting Phase 3 clinical trials for this investigational drug. Takeda is responsible for the research, development, and potential commercialization of VPRIV for Gaucher Disease, Type 1.
VPRIV is in Phase 3 clinical development. It is an investigational drug for Gaucher Disease, Type 1, and has not been approved by the FDA. Two Phase 3 trials have been completed, but the drug remains under investigation and is not yet available for general use.
VPRIV has been studied in two completed Phase 3 trials. The first, NCT00430625, enrolled 25 patients with Gaucher Disease, Type 1. The second, NCT00635427, was an open-label extension study with 95 patients. Both trials were completed, and no active trials are currently ongoing.
VPRIV is also known as GA-GCB, which stands for Gene-Activated Human Glucocerebrosidase. The clinical trials for VPRIV refer to it as GA-GCB enzyme replacement therapy. This alternative name is used in the study titles and descriptions for the Phase 3 trials.