Overview
Velaglucerase alfa is a gene-activated human recombinant glucocerebrosidase used for the treatment of Type 1 Gaucher disease, caused by a deficiency of the lysosomal enzyme glucocerebrosidase. Additionally, Velaglucerase alfa has also been investigated for use in Type 3 Gaucher disease.
Background
Velaglucerase alfa is a gene-activated human recombinant glucocerebrosidase used for the treatment of Type 1 Gaucher disease, caused by a deficiency of the lysosomal enzyme glucocerebrosidase. Additionally, Velaglucerase alfa has also been investigated for use in Type 3 Gaucher disease.
Indication
Velaglucerase alfa is a hydrolytic lysosomal glucocerebroside-specific enzyme indicated for long-term enzyme replacement therapy for pediatric and adult patients with type 1 Gaucher disease.
Associated Conditions
- Gaucher Disease, Type 1
Clinical Trials
Title | Posted | Study ID | Phase | Status | Sponsor |
|---|---|---|---|---|---|
2022/09/07 | Phase 3 | Completed | |||
2019/10/09 | Phase 4 | Completed | |||
2018/10/11 | Phase 4 | Completed | |||
2015/10/12 | Phase 4 | Completed | |||
2015/08/19 | Phase 4 | Withdrawn | |||
2013/04/30 | Phase 3 | Completed | |||
2009/08/07 | N/A | APPROVED_FOR_MARKETING | |||
2006/10/24 | Phase 1 | Completed |
FDA Drug Approvals
Approved Product | Manufacturer | NDC Code | Route | Strength | Effective Date |
|---|---|---|---|---|---|
| Takeda Pharmaceuticals America, Inc. | 54092-701 | INTRAVENOUS | 2.5 mg in 1 mL | 9/30/2021 |
EMA Drug Approvals
Approved Product | Authorization Holder | Status | Issued Date |
|---|---|---|---|
Authorised | 8/26/2010 | ||
Authorised | 8/26/2010 |
HSA Drug Approvals
Approved Product | Manufacturer | Approval Number | Dosage Form | Strength | Approval Date |
|---|---|---|---|---|---|
| VPRIV POWDER FOR SOLUTION FOR INFUSION 400 UNITS/VIAL | SIN16107P | INJECTION, POWDER, LYOPHILIZED, FOR SOLUTION | 10 mg/vial | 2/24/2021 |
NMPA Drug Approvals
Approved Product | Company | Approval Number | Drug Type | Dosage Form | Approval Date |
|---|---|---|---|---|---|
| Velaglucerase Alfa for Injection | 国药准字SJ20210013 | 生物制品 | 注射剂 | 4/27/2021 |
PPB Drug Approvals
Approved Product | Registration No. | Company | Licence No. | Strength | Registration Date |
|---|---|---|---|---|---|
| No PPB approvals found for this drug. | |||||
TGA Drug Approvals
Approved Product | ARTG ID | Sponsor | Registration Type | Status | Registration Date |
|---|---|---|---|---|---|
| VPRIV velaglucerase alfa (ghu) 400 Units powder for solution for infusion, glass vial | 180965 | Medicine | A | 2/29/2012 |