Substance / Medication

Olipudase alfa

Overview

Active Ingredient
olipudase alfa
RxNorm CUI
2610407

Indications

XENPOZYME is indicated for treatment of non–central nervous system manifestations of acid sphingomyelinase deficiency (ASMD) in adult and pediatric patients.

Labeler: Genzyme CorporationUpdated: 2025-12-11T00:00:00.000ZFull label on DailyMed

Boxed Warning

FDA Black Box Warning

Warnings and Precautions (5.1) [see] Patients treated with XENPOZYME have experienced life-threatening hypersensitivity reactions, including anaphylaxis. Appropriate medical monitoring and support measures, including cardiopulmonary resuscitation equipment, should be readily available during XENPOZY

Contraindications

When this intervention should not be used

None.

Indications & Related Conditions

Conditions associated via SNOMED clinical relationships

Administration & Protocol

Dosing, route, and treatment protocol

Detailed dosage and administration information is available in the full FDA drug label.

View full prescribing information on DailyMed

Monitoring & Follow-Up

Biomarkers relevant to this intervention via related conditions

No monitoring biomarkers have been mapped yet. Biomarker-intervention linkages are derived through related conditions and will expand as the knowledge graph grows.

Research Evidence

Published studies and systematic reviews

Sort:
Efficacy and Safety of Olipudase Alfa for the Treatment of Acid Sphingomyelinase Deficiency (ASMD): A Systematic Review and Meta-Analysis.
Antonello Breno Bopp, Giovacchini Giovanna, Albuquerque Anna Luiza Braga et al. · Am J Med Genet A · 2026
PMID: 40974024Meta-Analysis
The burden of rare damaging variants in hereditary atypical parkinsonism genes is increased in patients with Parkinson's disease.
Kim Yun Joong, Lee Jinwoo, Kim Nan Young et al. · Neurobiol Aging · 2021
PMID: 33423827Meta-Analysis
Lysosomal storage disorders as an etiology of nonimmune hydrops fetalis: A systematic review.
Iyer Neel S, Gimovsky Alexis C, Ferreira Carlos R et al. · Clin Genet · 2021
PMID: 34057202Meta-AnalysisFull text (PMC)
The natural history of Type 1 infantile GM1 gangliosidosis: A literature-based meta-analysis.
Lang Frederick M, Korner Paul, Harnett Mark et al. · Mol Genet Metab · 2020
PMID: 31937438Meta-AnalysisFull text (PMC)
Olipudase alfa for treatment of acid sphingomyelinase deficiency (ASMD): safety and efficacy in adults treated for 30 months.
Wasserstein Melissa P, Diaz George A, Lachmann Robin H et al. · J Inherit Metab Dis · 2018
PMID: 29305734TrialFull text (PMC)
Clearance of Hepatic Sphingomyelin by Olipudase Alfa Is Associated With Improvement in Lipid Profiles in Acid Sphingomyelinase Deficiency.
Thurberg Beth L, Wasserstein Melissa P, Jones Simon A et al. · Am J Surg Pathol · 2016
PMID: 27340749TrialFull text (PMC)

Research data from MEDLINE/PubMed

Benefits & Expected Outcomes

Benefits, expected outcomes, efficacy data, and NNT (Number Needed to Treat) are pending physician authorship and evidence review.

Risks & Side Effects

Adverse reaction and safety data for this drug is sourced from the FDA-approved label.

View adverse reactions & drug interactions on DailyMed

Related Symptoms

Symptoms associated with conditions this intervention addresses

No related symptoms have been mapped yet. Symptom linkages are derived through associated conditions.

Alternatives & Comparisons

Alternative treatments, comparison data, and clinical decision support are pending physician authorship.

Medical Disclaimer

This information is for educational purposes only and does not constitute medical advice, diagnosis, or treatment. Always consult with a qualified healthcare provider regarding any medical condition or treatment plan.

Do not start, stop, or change any treatment without consulting your healthcare provider.

Quick Facts

Type
Substance / Medication
Fully Specified Name
Olipudase alfa (substance)
SNOMED CT
2054161000122108
UMLS CUI
C4278198
RxNorm CUI
2610407
Labeler
Genzyme Corporation

Clinical Data

This intervention maps to 1 entities in the Ltrl knowledge graph.

1
Conditions
0
Biomarkers
0
Specialists
0
Symptoms

Data is sourced from SNOMED CT, UMLS, and the Ltrl clinical knowledge graph. Content sections marked as pending require physician authorship. Consult a healthcare provider before starting any treatment.