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Label indication VPRIV is indicated for long-term enzyme replacement therapy (ERT) for patients with type 1 Gaucher disease. VPRIV is a hydrolytic lysosomal glucocerebroside-specific enzyme indicated for long-term enzyme replacement therapy (ERT) for patients with type 1 Gaucher disease. ( 1 ) FDA label
Also known as GA-GCB (velaglucerase alfa), gene-activated® human glucocerebrosidase, VPRIV®, velaglucerase alfa, gene-activated glucocerebrosidase, DRX008, Enzyme replacement therapy
GA-GCB · 7 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.
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.
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.
Overall Summary of Treatment-emergent Adverse Events-Safety Population
| 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 | - |
| GA-GCB (velaglucerase alfa) | EXPERIMENTAL | 15-60 U/kg, every other week via intravenous infusion |
| 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 |
| 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: * 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 ...
GA-GCB is being developed for the treatment of Gaucher disease, a rare inherited disorder. It is an enzyme replacement therapy studied in patients with Type 1 Gaucher disease, including patients who had previously been treated with imiglucerase. Development is at Phase 2.
GA-GCB is being developed by Takeda Pharmaceutical Company Limited, which trades under the ticker TAK. Takeda is the sponsor behind the clinical program evaluating GA-GCB as an enzyme replacement therapy for Type 1 Gaucher disease.
GA-GCB is in Phase 2 development. It is an investigational enzyme replacement therapy and has not been approved. Two clinical trials of GA-GCB have been completed, and no trials are currently active.
Two completed trials have evaluated GA-GCB. NCT00478647 was a Phase 2 study in Type 1 Gaucher disease patients previously treated with imiglucerase, enrolling 40 patients across the United States, Israel, Poland, Spain, and the United Kingdom. NCT00391625 was a Phase 1 open-label extension study of long-term safety, enrolling 10 patients in Israel, Romania, and Serbia.
Yes. GA-GCB is also known as velaglucerase alfa. Both names refer to the same enzyme replacement therapy candidate being developed by Takeda for Type 1 Gaucher disease, so readers searching either name are looking at the same asset.
Across the two completed GA-GCB trials, 50 patients have been enrolled in total. NCT00478647 enrolled 40 patients with Type 1 Gaucher disease, and NCT00391625 enrolled 10 patients in an open-label extension study evaluating long-term safety.