Substance / Medication

Pegunigalsidase alfa

Overview

Active Ingredient
pegunigalsidase alfa
RxNorm CUI
2637446

Indications

ELFABRIO is indicated for the treatment of adults with confirmed Fabry disease.

Labeler: Chiesi USA, Inc.Updated: 2024-07-08T00:00:00.000ZFull label on DailyMed

Boxed Warning

FDA Black Box Warning

Patients treated with ELFABRIO have experienced hypersensitivity reactions, including anaphylaxis. Appropriate medical support measures, including cardiopulmonary resuscitation equipment, should be readily available during ELFABRIO administration. If a severe hypersensitivity reaction (e.g., anaphyl

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:
Long-term safety and efficacy of pegunigalsidase alfa: A multicenter 6-year study in adult patients with Fabry disease.
Hughes Derralynn, Gonzalez Derlis, Maegawa Gustavo et al. · Genet Med · 2023
PMID: 37634127Observational
A rapid method to reduce drug interferences for antibody measurements in pegunigalsidase alfa-treated patients with Fabry disease.
Lenders Malte, Rudolph Elisa, Rudnicki Michael et al. · Front Immunol · 2025
PMID: 41607773OtherFull text (PMC)
Health State Utility Values in Fabry Disease: Insights from the Pegunigalsidase Alfa Clinical Trials.
Azimpour Khashayar, Dorling Patricia, Koulinska Irene et al. · Adv Ther · 2025
PMID: 39847314OtherFull text (PMC)
Relevance of Neutralizing Antibodies for the Pharmacokinetics of Pegunigalsidase Alfa in Patients with Fabry Disease.
Lenders Malte, Menke Elise Raphaela, Rudnicki Michael et al. · BioDrugs · 2025
PMID: 39614966OtherFull 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
Pegunigalsidase alfa (substance)
SNOMED CT
2054151000122106
UMLS CUI
C5139798
RxNorm CUI
2637446
Labeler
Chiesi USA, 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.