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Elfabrio (PEGUNIGALSIDASE ALFA)
Elfabrio works by reducing the accumulation of a specific lipid in the body.
At a glance
| Generic name | PEGUNIGALSIDASE ALFA |
|---|---|
| Sponsor | Chiesi Farmaceutici S.P.A |
| Drug class | Hydrolytic Lysosomal Neutral Glycosphingolipid-specific Enzyme [EPC] |
| Modality | Enzyme |
| Therapeutic area | Metabolic |
| Phase | FDA-approved |
| First approval | 2023 |
Mechanism of action
Fabry disease is caused by deficiency of the lysosomal enzyme 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).
Approved indications
- Fabry's disease
Boxed warnings
- WARNING: HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS 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., anaphylaxis) occurs, discontinue ELFABRIO immediately and initiate appropriate medical treatment. In patients with severe hypersensitivity reaction, a desensitization procedure to ELFABRIO may be considered [see Warnings and Precautions ( 5.1 )] . WARNING: HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS See full prescribing information for complete boxed warning. Appropriate medical support measures, including cardiopulmonary resuscitation equipment, should be readily available. If a severe hypersensitivity reaction occurs, discontinue ELFABRIO immediately and initiate appropriate medical treatment. ( 5.1 )
Common side effects
- Infusion-associated reaction
- Nasopharyngitis
- Headache
- Diarrhea
- Fatigue
- Nausea
- Back pain
- Pain in extremity
- Sinusitis
- Abdominal Pain
- Proteinuria
- Hypersensitivity
Key clinical trials
- Dose-ranging Study of PRX-102 in Adult Fabry Disease Patients (PHASE1,PHASE2)
- Open Label Extension Study of 1 mg/kg Pegunigalsidase Alfa Every 2 Weeks in Patients With Fabry Disease (PHASE3)
- A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents With Fabry Disease (PHASE2,PHASE3)
- 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
- Study to Evaluate the Safety, PK, PD, and Efficacy of PRX-102 in Japanese Patients With Fabry Disease (PHASE2,PHASE3)
- Observational Study on Long-term Use of Pegunigalsidase Alfa in Fabry Patients in a Real-world Setting
- Open Label Extension of 2 mg/kg Pegunigalsidase Alfa (PRX-102) Every 4 Weeks in Adult Fabry Disease Patients (PHASE3)
- A Multi-Country Observational Study of Safety and Effectiveness of Elfabrio® in Fabry Patients
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| FDA label | Mechanism, indications, dosing, boxed warnings, drug interactions |
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- Elfabrio CI brief — competitive landscape report
- Elfabrio updates RSS · CI watch RSS
- Chiesi Farmaceutici S.P.A portfolio CI