Olipudase alfa is a recombinant human acid sphingomyelinase used to treat Acid Sphingomyelinase Deficiency (ASMD) in children and adults.

Generic Name
Olipudase alfa
DrugBank Accession Number

Olipudase alfa is recombinant human acid sphingomyelinase.1 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.6 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.3 Olipudase alfa works to hydrolyze sphingomyelin accumulated in body tissues, such as the lungs, liver, spleen, kidneys, and bone marrow.1

Olipudase alfa gained its first global approval in Japan on March 28, 2022.1 It was later approved by the European Commission on June 28, 2022 6 and by the FDA on August 31, 2022.8

Approved, Investigational
Biologic Classification
Protein Based Therapies
Other protein based therapies
Protein Chemical Formula
Protein Average Weight
63590.933 Da (exact)
>Olipudase alfa sequence
  1. KEGG DRUG: Olipudase alfa [Link]
Download FASTA Format
  • Olipudase alfa
External IDs
  • Gz402665



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.5,7

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Associated Conditions
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Olipudase alfa is an enzyme replacement therapy that works to ameliorate the signs and symptoms of ASMD by reducing the amount of sphingomyelin that accumulates in organs and causes tissue damage in patients with ASMD.5 It works to reduce the extent of non-neurological manifestations of ASMD, such as splenomegaly and hepatomegaly.4

Mechanism of action

ASMD is an autosomal recessive genetic disorder caused by different mutations in the SMPD1 gene that encodes acid sphingomyelinase. Historically, ASMD has been called Niemann-Pick disease (NPD), with different classifications based on disease onset and severity.3 NPD type A (NPD-A) refers to the severe early-onset form, infantile neurovisceral ASMD and NPD type B (NPD-B) is referred to as the later-onset, chronic visceral form of ASMD. Chronic neurovisceral ASMD (NPD-A/B) is a phenotype with intermediate severity.2 ASMD has a broad spectrum of disease severity and neurological and non-neurological manifestations; thus, it is difficult to classify different types of ASMD using the former classification system of ASMD (A, B, A/B).3 Acid sphingomyelinase typically breaks down metabolically-related lipids such as sphingomyelin in various cell types, such as the monocytes, macrophages, and hepatocytes.3 The deficiency of acid sphingomyelinase thus leads to the accumulation of these lipids in body tissues, causing progressive cell and tissue damage and impairing organ functioning.3 Olipudase alfa is recombinant human acid sphingomyelinase that hydrolyzes sphingomyelin (SM), preventing its accumulation in body organs. As an enzyme replacement therapy, it is an exogenous source of acid sphingomyelinase.5


After administration of 3 mg/kg olipudase alfa once every two weeks in adults with ASMD, the mean (percent coefficient of variation, CV%) maximum concentration (Cmax) and area under the concentration-time curve over a dosing interval (AUC0-τ) at steady state were 30.2 µg/mL (17%) and 607 µg.h/mL (20%), respectively.5

Volume of distribution

After administration of 3 mg/kg olipudase alfa once every two weeks in adults with ASMD, the estimated mean (CV%) volume of distribution of olipudase alfa is 13.1 L (18%). Olipudase alfa is not expected to cross the blood-brain barrier or modulate the CNS manifestations of the disease.5

Protein binding

There is no information available.


Olipudase alfa is a recombinant human enzyme and is expected to be eliminated via proteolytic degradation into small peptides and amino acids.5

Route of elimination

There is no information available.


After administration of 3 mg/kg olipudase alfa once every two weeks in adults with ASMD, the mean terminal half-life (t1/2) ranged from 31.9 to 37.6 hours.5


After administration of 3 mg/kg olipudase alfa once every two weeks in adults with ASMD, the mean (CV%) clearance of olipudase alfa is 0.331 L/h (22%).5

Adverse Effects
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There is no information available regarding LD50 and overdose of olipudase alfa. There is no known specific antidote for olipudase alfa overdose.

Not Available
Pharmacogenomic Effects/ADRs
Not Available


Drug Interactions
This information should not be interpreted without the help of a healthcare provider. If you believe you are experiencing an interaction, contact a healthcare provider immediately. The absence of an interaction does not necessarily mean no interactions exist.
Not Available
Food Interactions
No interactions found.


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Brand Name Prescription Products
NameDosageStrengthRouteLabellerMarketing StartMarketing EndRegionImage
XenpozymeInjection, powder, for solution20 mgIntravenousGenzyme Europe Bv2022-08-02Not applicableEU flag
XenpozymeInjection, powder, for solution20 mgIntravenousGenzyme Europe Bv2022-08-02Not applicableEU flag
XenpozymeInjection, powder, lyophilized, for solution20 mg/5.1mLIntravenousGenzyme Corporation2022-08-31Not applicableUS flag
XenpozymeInjection, powder, for solution20 mgIntravenousGenzyme Europe Bv2022-08-02Not applicableEU flag
XenpozymeInjection, powder, for solution20 mgIntravenousGenzyme Europe Bv2022-08-02Not applicableEU flag


Drug Categories
Chemical TaxonomyProvided by Classyfire
Not Available
Organic Compounds
Super Class
Organic Acids
Carboxylic Acids and Derivatives
Sub Class
Amino Acids, Peptides, and Analogues
Direct Parent
Alternative Parents
Not Available
Not Available
Molecular Framework
Not Available
External Descriptors
Not Available
Affected organisms
Not Available

Chemical Identifiers

CAS number


General References
  1. Keam SJ: Olipudase Alfa: First Approval. Drugs. 2022 Jun;82(8):941-947. doi: 10.1007/s40265-022-01727-x. [Article]
  2. Wasserstein MP, Schuchman EH: Acid Sphingomyelinase Deficiency . [Article]
  3. McGovern MM, Avetisyan R, Sanson BJ, Lidove O: Disease manifestations and burden of illness in patients with acid sphingomyelinase deficiency (ASMD). Orphanet J Rare Dis. 2017 Feb 23;12(1):41. doi: 10.1186/s13023-017-0572-x. [Article]
  4. Diaz GA, Jones SA, Scarpa M, Mengel KE, Giugliani R, Guffon N, Batsu I, Fraser PA, Li J, Zhang Q, Ortemann-Renon C: One-year results of a clinical trial of olipudase alfa enzyme replacement therapy in pediatric patients with acid sphingomyelinase deficiency. Genet Med. 2021 Aug;23(8):1543-1550. doi: 10.1038/s41436-021-01156-3. Epub 2021 Apr 19. [Article]
  5. EMA Approved Drug Products: Xenpozyme (olipudase alfa) Intravenous Infusion [Link]
  6. GlobeNewswire News Release: Xenpozyme® (olipudase alfa) approved by European Commission as first and only treatment for ASMD [Link]
  7. FDA Approved Drug Products: XENPOZYME (olipudase alfa-rpcp) for injection, for intravenous use [Link]
  8. FDA NEWS RELEASE: FDA Approves First Treatment for Acid Sphingomyelinase Deficiency, a Rare Genetic Disease [Link]
PubChem Substance

Clinical Trials

Clinical Trials
2Active Not RecruitingTreatmentSphingomyelin Lipidosis1
2, 3Active Not RecruitingTreatmentSphingomyelin Lipidosis1
1CompletedTreatmentAcid Sphingomyelinase Deficiency (ASMD)1
1, 2CompletedTreatmentSphingomyelin Lipidosis1
Not AvailableApproved for MarketingNot AvailableSphingomyelin Lipidosis1
Not AvailableRecruitingNot AvailableAcid Sphingomyelinase Deficiency (ASMD)1


Not Available
Not Available
Dosage Forms
Injection, powder, for solutionIntravenous20 mg
Injection, powder, lyophilized, for solutionIntravenous20 mg/5.1mL
Not Available
Not Available


Experimental Properties
Not Available

Drug created at October 21, 2016 00:36 / Updated at September 15, 2022 21:58