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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 VPRIV, Gene activated glucocerebrosidase, GA-GCB, Enzyme replacement therapy, Gene activated human glucocerebrosidase, Gene-Activated® Human Glucocerebrosidase(GA-GCB), DRX008
velaglucerase alfa · 9 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 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
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 |
| 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 |
| GA-GCB | EXPERIMENTAL | VPRIV™ ,velaglucerase alfa |
| imiglucerase | ACTIVE_COMPARATOR | - |
| 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) |
| 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. |
| VPRIV® | BIOLOGICAL | Intravenous infusion, every other week (EOW) |
| imiglucerase | BIOLOGICAL | IV infusion, 60 U/kg every other week for 9 months |
| VPRIV ®, | BIOLOGICAL | Intravenous (IV) infusion, every other week via intravenous infusion for 12 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 ...
Velaglucerase alfa is used to treat Gaucher disease, including Type 1 and Type 3 forms of the condition. It is studied in both children and adults, with clinical trials enrolling patients as young as 2 years of age. Gaucher disease is a rare inherited disorder, and velaglucerase alfa is given as an enzyme replacement therapy.
Velaglucerase alfa is developed by Takeda Pharmaceutical Company Limited, which trades under the ticker TAK. Takeda is the sponsor behind the clinical development program for the drug across its Gaucher disease indications.
Velaglucerase alfa has been studied in Phase 3 clinical trials, with additional Phase 1 research also conducted. Its trials in Gaucher disease are listed as completed, and no trials are currently active. The program spans Type 1 and Type 3 Gaucher disease across multiple countries.
Velaglucerase alfa has been evaluated in completed trials including NCT05529992, a Phase 3 study in Chinese patients with Type 1 Gaucher disease, NCT01842841, a Phase 3 extension study in Japanese patients, NCT01685216, a Phase 1 study in children and adolescents with Type 3 Gaucher disease, and NCT01614574, a Phase 3 study in Japanese patients.
Across its listed clinical trials, velaglucerase alfa enrolled a total of 34 patients. Individual studies ranged from 5 to 20 participants. The trials were conducted in countries including China, Japan, Egypt, India, and Tunisia, and all enrolled patients were 2 years of age or older.