BLASINGLE-USEVIALPriority Review
Approved
May 2023
Lifecycle
Growth
Competitive Pressure
30/100
Clinical Trials
7
Data completeness
5/8 — Partial
Priority Review
ELFABRIO (pegunigalsidase alfa) by Chiesi is alpha-galactosidase a. First approved in 2023.
Drug data last refreshed 2h ago · AI intelligence enriched 1w ago
AI summarySynthesized from FDA + DailyMed + ClinicalTrials.gov data
ELFABRIO (pegunigalsidase alfa) is a pegylated recombinant alpha-galactosidase A enzyme replacement therapy approved in May 2023 for Fabry disease. It works by providing an exogenous source of alpha-galactosidase A that is internalized into lysosomes to reduce accumulated globotriaosylceramide (Gb3), the pathogenic substrate in this lysosomal storage disorder. ELFABRIO is administered as a single-use intravenous infusion.
$0.47MPart D
GrowthEarly-stage rare disease product with modest current penetration; brand team is building market awareness and clinical adoption in a niche, high-value patient population.
Mechanism of Action
alpha-galactosidase A. ELFABRIO provides an exogenous source of alpha-galactosidase A. ELFABRIO is internalized and transported into lysosomes where it is thought to exert enzymatic activity and reduce accumulated globotriaosylceramide (Gb3).
Pharmacologic Class:
Hydrolytic Lysosomal Neutral Glycosphingolipid-specific Enzyme
Indications
Indication data is being enriched from DailyMed and FDA labeling. Check back soon for approved therapeutic uses.
Clinical Trials (7)
Observational Study on Long-term Use of Pegunigalsidase Alfa in Fabry Patients in a Real-world Setting
Started Apr 2026
75 enrolled
Fabry Disease
Maternal and Postnatal Outcomes Study (MOS): A Global Observational Registry Assessing the Safety of Elfabrio® in Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding
Started May 2025
10 enrolled
Fabry DiseasePregnancyPregnancy Complications
A Multi-Country Observational Study of Safety and Effectiveness of Elfabrio® in Fabry Patients
Started Nov 2024
100 enrolled
Fabry Disease
Fabry Patient's Experience Of PegunigaLsidasE Alfa Monthly Infusion
Started Jan 2022
23 enrolled
Fabry Disease
Open Label Extension of 2 mg/kg Pegunigalsidase Alfa (PRX-102) Every 4 Weeks in Adult Fabry Disease Patients
Started Nov 2018
29 enrolled
Fabry Disease
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Data Sources
- Drug label: DailyMed / FDA
- Clinical trials: ClinicalTrials.gov
- Patent data: FDA Orange Book
- Spending data: CMS Medicare
The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.