Substance / Medication

Elosulfase alfa

Overview

Active Ingredient
elosulfase alfa
RxNorm CUI
1489914

Indications

VIMIZIM (elosulfase alfa) is indicated for patients with Mucopolysaccharidosis type IVA (MPS IVA; Morquio A syndrome).

Labeler: BioMarin Pharmaceutical Inc.Updated: 2025-10-30T00:00:00.000ZFull label on DailyMed

Boxed Warning

FDA Black Box Warning

Patients treated with enzyme replacement therapies have experienced life-threatening hypersensitivity reactions, including anaphylaxis. Anaphylaxis has occurred during the early course of enzyme replacement therapy and after extended duration of therapy. Warnings and Precautions (5.1) [see] Initiate

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:
Successful Elosulfase Alfa Desensitization Protocol in a Patient With Morquio A Syndrome.
Díaz Vidal Verónica C, Gillispie Amanda, Aranda Carolina et al. · Pediatrics · 2022
PMID: 35059725Case Report
Shortened desensitization leading to a 2-year enzyme replacement therapy with elosulfase alfa.
Bekis Bozkurt Hayrunnisa, Karakurt Tuba, Cavkaytar Ozlem et al. · Ann Allergy Asthma Immunol · 2021
PMID: 33992778Case Report
Clinical Utility of Elosulfase Alfa in the Treatment of Morquio A Syndrome.
Lee Chung-Lin, Chuang Chih-Kuang, Chiu Huei-Ching et al. · Drug Des Devel Ther · 2022
PMID: 35046639ReviewFull text (PMC)
Long-term outcomes of elosulfase alfa enzyme replacement therapy in adults with MPS IVA: a sub-analysis of the Morquio A Registry Study (MARS).
Stepien Karolina M, Burton Barbara K, Bober Michael B et al. · Orphanet J Rare Dis · 2025
PMID: 41168830OtherFull text (PMC)
Real-world treatment with elosulfase alfa in patients with MPS IVA is associated with improved endurance over time.
Burton Barbara K, Stepien Karolina M, Campeau Philippe M et al. · Genet Med Open · 2025
PMID: 40677308OtherFull text (PMC)
Efficacy of Intravenous Elosulfase Alfa for Mucopolysaccharidosis Type IVA: A Systematic Review and Meta-Analysis.
Lee Chung-Lin, Chuang Chih-Kuang, Syu Yu-Min et al. · J Pers Med · 2022
PMID: 36013287OtherFull 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
Elosulfase alfa (substance)
SNOMED CT
702395005
UMLS CUI
C3817397
RxNorm CUI
1489914
Labeler
BioMarin Pharmaceutical Inc.

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.