Overview
Olipudase alfa is recombinant human acid sphingomyelinase. It is the first and only enzyme replacement therapy in the world for the treatment of Acid Sphingomyelinase Deficiency (ASMD), also known as Niemann–Pick disease. ASMD is a rare lysosomal storage disease caused by mutations in the SMPD1 gene, leading to a deficiency in acid sphingomyelinase and the abnormal accumulation of the primary ASM substrate, sphingomyelin. Olipudase alfa works to hydrolyze sphingomyelin accumulated in body tissues, such as the lungs, liver, spleen, kidneys, and bone marrow. Olipudase alfa gained its first global approval in Japan on March 28, 2022. It was later approved by the European Commission on June 28, 2022 and by the FDA on August 31, 2022.
Background
Olipudase alfa is recombinant human acid sphingomyelinase. It is the first and only enzyme replacement therapy in the world for the treatment of Acid Sphingomyelinase Deficiency (ASMD), also known as Niemann–Pick disease. ASMD is a rare lysosomal storage disease caused by mutations in the SMPD1 gene, leading to a deficiency in acid sphingomyelinase and the abnormal accumulation of the primary ASM substrate, sphingomyelin. Olipudase alfa works to hydrolyze sphingomyelin accumulated in body tissues, such as the lungs, liver, spleen, kidneys, and bone marrow. Olipudase alfa gained its first global approval in Japan on March 28, 2022. It was later approved by the European Commission on June 28, 2022 and by the FDA on August 31, 2022.
Indication
Olipudase alfa is indicated as an enzyme replacement therapy for the treatment of non-Central Nervous System manifestations of Acid Sphingomyelinase Deficiency (ASMD) in pediatric and adult patients with type A/B or type B.
Associated Conditions
- Acid Sphingomyelinase Deficiency (ASMD)
Clinical Trials
Title | Posted | Study ID | Phase | Status | Sponsor |
---|---|---|---|---|---|
2025/04/29 | Phase 2 | Completed | |||
2024/01/05 | N/A | Recruiting | |||
2022/05/03 | N/A | Completed | |||
2014/11/17 | Phase 1 | Completed | Genzyme, a Sanofi Company | ||
2013/12/09 | Phase 2 | Completed | Genzyme, a Sanofi Company | ||
2013/12/09 | Phase 2 | Completed | Genzyme, a Sanofi Company | ||
2006/12/13 | Phase 1 | Terminated | Genzyme, a Sanofi Company |
FDA Drug Approvals
Approved Product | Manufacturer | NDC Code | Route | Strength | Effective Date |
---|---|---|---|---|---|
Genzyme Corporation | 58468-0050 | INTRAVENOUS | 20 mg in 5.1 mL | 12/12/2023 | |
Genzyme Corporation | 58468-0051 | INTRAVENOUS | 4 mg in 1.1 mL | 12/12/2023 |
EMA Drug Approvals
Approved Product | Authorization Holder | Status | Issued Date |
---|---|---|---|
Authorised | 6/24/2022 | ||
Authorised | 6/24/2022 | ||
Authorised | 6/24/2022 | ||
Authorised | 6/24/2022 | ||
Authorised | 6/24/2022 | ||
Authorised | 6/24/2022 |
HSA Drug Approvals
Approved Product | Manufacturer | Approval Number | Dosage Form | Strength | Approval Date |
---|---|---|---|---|---|
No HSA approvals found for this drug. |
NMPA Drug Approvals
Approved Product | Company | Approval Number | Drug Type | Dosage Form | Approval Date |
---|---|---|---|---|---|
No NMPA approvals found for this drug. |
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 |
---|---|---|---|---|---|
XENPOZYME olipudase alfa 20 mg powder for injection vial | 394103 | Medicine | A | 8/24/2023 | |
XENPOZYME olipudase alfa 4 mg powder for injection vial | 423370 | Medicine | A | 5/3/2024 |