Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Velaglucerase Alfa · 5 trials · 3 indications
Adverse event(AE)=any untoward medical occurrence in clinical investigation participant administered a drug;it does not necessarily have to have causal relationship with this treatment. AE can therefore be any unfavorable\&unintended sign (example,clinically significant abnormal laboratory value),symptom/disease temporally associated with use of drug whether or not it is considered related to drug. TEAE=any event emerging or manifesting at or after initiation of investigational product or any existing event that worsens in either intensity or frequency following exposure to investigational product. SAE=any untoward clinical manifestation of signs, symptoms, or outcomes(whether considered related to investigational product or not)\&at any dose: results in death,is life-threatening,requires in-patient hospitalization/prolongation of hospitalization,results in persistent/significant disability/incapacity,results in congenital abnormality/birth defect,or is an important medical event.
An AE was any noxious, pathologic, or unintended change in anatomical, physiologic, or metabolic function as indicated by physical signs, symptoms, or laboratory changes occurring in any phase of a clinical study, whether or not considered related to investigational product. A SAE was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged in-patient hospitalization; life threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. An infusion-related AE was defined as an AE that started either during or within 12 hours after the start of the infusion and that was judged as possibly or probably related to investigational product.
Laboratory test results were considered abnormal and clinically significant at the discretion of the investigator.
Serum samples were collected for all participants for determination of anti-velaglucerase alfa antibodies every 12 weeks.
Hemoglobin concentration was measured as part of the hematology panel or measured separately when the hematology panel was not scheduled. Samples were measured by a central laboratory. Baseline is the modified baseline hemoglobin concentration, the average of the values from screening, baseline, and Week 1/Day 1. A positive change from baseline indicates that hemoglobin concentration increased.
| Arm | Type | Description |
|---|---|---|
| Velaglucerase Alfa (VPRIV) | EXPERIMENTAL | Participants received VPRIV IV infusion at 60 units per kilogram (U/kg) body weight once every other week (EOW) for 60 (+10) minutes for up to 51 weeks. |
| velaglucerase alfa | EXPERIMENTAL | 15 to 60 U/kg, EOW via intravenous infusion |
| Investigational | EXPERIMENTAL | velaglucerase alfa |
| GA-GCB | EXPERIMENTAL | VPRIV™ ,velaglucerase alfa |
| imiglucerase | ACTIVE_COMPARATOR | - |
| Name | Type | Description |
|---|---|---|
| Velaglucerase Alfa | DRUG | VPRIV intravenous infusion every other week for 60 minutes. |
| imiglucerase | BIOLOGICAL | IV infusion, 60 U/kg every other week for 9 months |
Inclusion: * Has a documented, confirmed diagnosis of type 1 Gaucher disease based on the following, as determined by the investigator: 1. Decreased glucocerebrosidase (GCB) activity level that is ≤30% of normal or 2. Decreased GCB activity level that is \>30% of normal, but with confirmation ...
Velaglucerase Alfa is an investigational enzyme replacement therapy being studied for the treatment of Gaucher Disease, including Type 1 and Type 3. It is developed by Takeda Pharmaceutical Company Limited (TAK) and is currently in Phase 3 clinical development for these rare genetic conditions.
Velaglucerase Alfa is a small molecule designed to replace the deficient enzyme glucocerebrosidase in patients with Gaucher Disease. By providing this enzyme, it aims to reduce the accumulation of glucocerebroside in cells, which is the underlying cause of the disease's symptoms.
Velaglucerase Alfa is developed by Takeda Pharmaceutical Company Limited, a global biopharmaceutical company. Takeda is listed on the stock exchange under the ticker symbol TAK.
Velaglucerase Alfa is in Phase 3 clinical development. It is not yet approved by regulatory authorities and remains an investigational drug. Several Phase 3 trials have been completed, but the drug is still undergoing clinical evaluation for Gaucher Disease.
Velaglucerase Alfa has been studied in several clinical trials, including NCT00553631, a Phase 3 study comparing it with imiglucerase in Type 1 Gaucher Disease, and NCT01685216, a Phase 1 study in children with Type 3 Gaucher Disease. Other trials include NCT01842841 and NCT05529992, both Phase 3 studies in Japanese and Chinese patients, respectively.
Velaglucerase Alfa is also known as VPRIV. In clinical trials, such as NCT05529992, the drug is referred to as Velaglucerase Alfa (VPRIV), indicating that these names refer to the same investigational therapy for Gaucher Disease.