Olipudase alfa

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.

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
A Study to Evaluate Safety and Tolerability of Olipudase Alfa in Pediatric and Adult Participants With Acid Sphingomyelinase Deficiency (ASMD) Who Completed the DFI12712 or the LTS13632 Study in France	2025/04/29	NCT06949358	Phase 2	Completed	Sanofi
A Real-world Long-term Safety and Immunogenicity Study of Olipudase Alfa Therapy in Pediatric Patients Less Than 2 Years of Age With Acid Sphingomyelinase Deficiency (ASMD)	2024/01/05	NCT06192576	N/A	Recruiting	Sanofi
Data Analysis of Adult and Pediatric Participants With Acid Sphingomyelinase Deficiency (ASMD) on Early Access to Olipudase Alfa in France	2022/05/03	NCT05359276	N/A	Completed	Sanofi
Safety, Tolerability, PK, and Efficacy Evaluation of Repeat Ascending Doses of Olipudase Alfa in Pediatric Patients <18 Years of Age With Acid Sphingomyelinase Deficiency	2014/11/17	NCT02292654	Phase 1	Completed	Genzyme, a Sanofi Company
A Long-Term Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency	2013/12/09	NCT02004704	Phase 2	Completed	Genzyme, a Sanofi Company
Efficacy, Safety, Pharmacodynamic, and Pharmacokinetics Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency	2013/12/09	NCT02004691	Phase 2	Completed	Genzyme, a Sanofi Company
Safety Study of rhASM Enzyme Replacement Therapy in Adults With Acid Sphingomyelinase Deficiency (Niemann-Pick Disease)	2006/12/13	NCT00410566	Phase 1	Terminated	Genzyme, a Sanofi Company

FDA Drug Approvals

Approved Product	Manufacturer	NDC Code	Route	Strength	Effective Date
XENPOZYME	Genzyme Corporation	58468-0050	INTRAVENOUS	20 mg in 5.1 mL	12/12/2023
XENPOZYME	Genzyme Corporation	58468-0051	INTRAVENOUS	4 mg in 1.1 mL	12/12/2023

EMA Drug Approvals

Approved Product	Authorization Holder	Status	Issued Date
Xenpozyme	Sanofi B.V.,Paasheuvelweg 25,1105 BP Amsterdam,Netherlands	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	Sanofi-Aventis Australia Pty Ltd	Medicine	A	8/24/2023
XENPOZYME olipudase alfa 4 mg powder for injection vial	423370	Sanofi-Aventis Australia Pty Ltd	Medicine	A	5/3/2024

Health Canada Drug Approvals

Approved Product	Company	DIN	Dosage Form	Strength	Market Date
XENPOZYME	sanofi-aventis canada inc	02545713	Powder For Solution - Intravenous	4 MG / VIAL	N/A

CIMA AEMPS Drug Approvals

Approved Product	Company	Registration Number	Pharmaceutical Form	Prescription Type	Status
XENPOZYME 20 MG POLVO PARA CONCENTRADO PARA SOLUCION PARA PERFUSION	Sanofi B.V.	1221659001	POLVO PARA CONCENTRADO PARA SOLUCIÓN PARA PERFUSIÓN	Uso Hospitalario	Commercialized
XENPOZYME 4 MG POLVO PARA CONCENTRADO PARA SOLUCION PARA PERFUSION	Sanofi B.V.	1221659005	POLVO PARA CONCENTRADO PARA SOLUCIÓN PARA PERFUSIÓN	Uso Hospitalario	Commercialized

Philippines FDA Drug Approvals

Approved Product	Company	License Number	Dosage Form	Strength	Approval Date
No Philippines FDA approvals found for this drug.

Saudi SFDA Drug Approvals

Approved Product	Company	License Number	Dosage Form	Strength	Approval Date
No Saudi SFDA approvals found for this drug.

Malaysia NPRA Drug Approvals

Approved Product	Company	Registration Number	Dosage Form	Strength	Approval Date
No Malaysia NPRA approvals found for this drug.

UK EMC Drug Information

Medicine Name	MA Holder	MA Number	Pharmaceutical Form	Active Ingredient	Authorization Date
No UK EMC drug information found for this drug.

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