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Olipudase alfa

Generic Name
Olipudase alfa
Brand Names
Xenpozyme
Drug Type
Biotech
CAS Number
927883-84-9
Unique Ingredient Identifier
6D5766Q4OP

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)

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

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
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