BLASINGLE-USEVIALPriority Review
Approved
May 2023
Lifecycle
Growth
Competitive Pressure
30/100
Clinical Trials
7
Data completeness
6/8 — Strong
Priority Review
ELFABRIO (pegunigalsidase alfa) by Chiesi is alpha-galactosidase a. Approved for fabry disease. First approved in 2023.
Drug data last refreshed 19h ago · AI intelligence enriched 2w ago
AI summarySynthesized from FDA + DailyMed + ClinicalTrials.gov data
ELFABRIO (pegunigalsidase alfa) is a PEGylated recombinant alpha-galactosidase A enzyme approved for Fabry disease, a rare lysosomal storage disorder. The drug provides an exogenous enzyme source that is internalized into lysosomes where it reduces accumulated globotriaosylceramide (Gb3), the pathogenic substrate in Fabry disease. Administered as an intravenous infusion, ELFABRIO represents a next-generation enzyme replacement therapy with improved pharmacokinetics over earlier agents.
$0.47MPart D
GrowthEarly-stage growth phase product with modest current utilization but significant upside potential as awareness and diagnosis of Fabry disease expands in patient populations.
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 (1)
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.