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:
Impact of long-term elosulfase alfa on activities of daily living in patients with Morquio A syndrome in an open-label, multi-center, phase 3 extension study.
Hendriksz Christian J, Parini Rossella, AlSayed Moeenaldeen D et al. · Mol Genet Metab · 2018
PMID: 29248359Trial
Impact of elosulfase alfa in patients with morquio A syndrome who have limited ambulation: An open-label, phase 2 study.
Harmatz Paul R, Mengel Eugen, Geberhiwot Tarekegn et al. · Am J Med Genet A · 2017
PMID: 27774754TrialFull text (PMC)
Impact of long-term elosulfase alfa treatment on respiratory function in patients with Morquio A syndrome.
Hendriksz Christian J, Berger Kenneth I, Parini Rossella et al. · J Inherit Metab Dis · 2016
PMID: 27553181TrialFull text (PMC)
Long-term endurance and safety of elosulfase alfa enzyme replacement therapy in patients with Morquio A syndrome.
Hendriksz Christian J, Parini Rossella, AlSayed Moeenaldeen D et al. · Mol Genet Metab · 2016
PMID: 27380995Trial

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.