ELFABRIO
ELFABRIO (pegunigalsidase alfa-iwxj) is a hydrolytic lysosomal neutral glycosphingolipid-specific enzyme. It is indicated for the treatment of adults with a confirmed diagnosis of Fabry disease. The drug serves as a therapeutic replacement for patients who lack sufficient levels of the naturally occurring enzyme required to process specific lipids.
How ELFABRIO Works
Fabry disease is caused by a deficiency in the lysosomal enzyme alpha-galactosidase A. ELFABRIO provides an exogenous source of this enzyme, which is internalized and transported into the lysosomes of the cells. Once inside the lysosomes, the drug exerts enzymatic activity to reduce the accumulation of globotriaosylceramide (Gb3).
Details
- Status
- Prescription
- First Approved
- 2023-05-09
- Routes
- SINGLE-USE
- Dosage Forms
- VIAL
ELFABRIO Approval History
What ELFABRIO Treats
1 indicationsELFABRIO is approved for 1 conditions since its original approval in 2023. These indications span multiple therapeutic areas including oncology, immunology, and more.
- Fabry Disease
ELFABRIO Boxed 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 desensi...
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 )
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Active Pipeline
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Trial Timeline
Full development history with FDA approval milestones
Understanding FDA Approval Types
| Count | Type | What it means |
|---|---|---|
| - | ORIG | Original approval - drug first enters market |
| - | SUPPL - Efficacy | New indication (new disease/condition approved) |
| - | SUPPL - Labeling | Label text changes (warnings, dosing updates) |
| - | SUPPL - Manufacturing | Production changes (new facility) |
| - | SUPPL - Chemistry | Formulation changes (new dosage strength) |
Green lines in the timeline show ORIG and Efficacy approvals - the clinically meaningful milestones.
ELFABRIO FDA Label Details
ProIndications & Usage
FDA Label (PDF)ELFABRIO is indicated for the treatment of adults with confirmed Fabry disease. ELFABRIO is a hydrolytic lysosomal neutral glycosphingolipid-specific enzyme indicated for the treatment of adults with confirmed Fabry disease.
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 administra...
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Data Sources
Data sourced from official FDA and NIH databases. Click links to verify on original sources.