Last reviewed · How we verify
ELFABRIO (PEGUNIGALSIDASE ALFA-IWXJ)
ELFABRIO (generic name: PEGUNIGALSIDASE ALFA-IWXJ) is a Hydrolytic Lysosomal Neutral Glycosphingolipid-specific Enzyme [EPC] drug developed by CHIESI FARMACEUTICI SPA. It is currently FDA-approved for Fabry's disease.
At a glance
| Generic name | PEGUNIGALSIDASE ALFA-IWXJ |
|---|---|
| Sponsor | CHIESI FARMACEUTICI SPA |
| Drug class | Hydrolytic Lysosomal Neutral Glycosphingolipid-specific Enzyme [EPC] |
| Therapeutic area | Rare Disease |
| Phase | FDA-approved |
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 reactions
- Nasopharyngitis
- Headache
- Diarrhea
- Fatigue
- Nausea
- Back pain
- Pain in extremity
- Sinusitis
Serious adverse events
- Severe hypersensitivity reaction
- Membranoproliferative glomerulonephritis
- Anaphylaxis
Key clinical trials
- Maternal and Postnatal Outcomes Study (MOS) A Worldwide Decentralized Observational Registry to Evaluate the Safety in Women With Fabry Disease and Their Infants Exposed to Elfabrio® (Pegunigalsidase (N/A)
- A Multi-Country Observational Study of Safety and Effectiveness of Elfabrio® in Fabry Patients (N/A)
- Observational Prospective Cohort Study on Long-term Effective and Safe Use of Pegunigalsidase Alfa in Adult Fabry Patients Under "Real-world" Setting (N/A)
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 |
|---|---|
| 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 SPA portfolio CI
Frequently asked questions about ELFABRIO
What is ELFABRIO?
ELFABRIO (PEGUNIGALSIDASE ALFA-IWXJ) is a Hydrolytic Lysosomal Neutral Glycosphingolipid-specific Enzyme [EPC] drug developed by CHIESI FARMACEUTICI SPA, indicated for Fabry's disease.
What is ELFABRIO used for?
ELFABRIO is indicated for Fabry's disease.
Who makes ELFABRIO?
ELFABRIO is developed and marketed by CHIESI FARMACEUTICI SPA (see full CHIESI FARMACEUTICI SPA pipeline at /company/chiesi-farmaceutici-spa).
What is the generic name of ELFABRIO?
PEGUNIGALSIDASE ALFA-IWXJ is the generic (nonproprietary) name of ELFABRIO.
What drug class is ELFABRIO in?
ELFABRIO belongs to the Hydrolytic Lysosomal Neutral Glycosphingolipid-specific Enzyme [EPC] class. See all Hydrolytic Lysosomal Neutral Glycosphingolipid-specific Enzyme [EPC] drugs at /class/hydrolytic-lysosomal-neutral-glycosphingolipid-specific-enzyme-epc.
What development phase is ELFABRIO in?
ELFABRIO is FDA-approved (marketed).
What are the side effects of ELFABRIO?
Common side effects of ELFABRIO include Infusion-associated reactions, Nasopharyngitis, Headache, Diarrhea, Fatigue, Nausea. Serious adverse events: Severe hypersensitivity reaction, Membranoproliferative glomerulonephritis, Anaphylaxis.
Related
- Drug class: All Hydrolytic Lysosomal Neutral Glycosphingolipid-specific Enzyme [EPC] drugs
- Manufacturer: CHIESI FARMACEUTICI SPA — full pipeline
- Therapeutic area: All drugs in Rare Disease
- Indication: Drugs for Fabry's disease
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing