Elfabrio FDA Approval History
Last updated by Judith Stewart, BPharm on May 11, 2023.
FDA Approved: Yes (First approved May 9, 2023)
Brand name: Elfabrio
Generic name: pegunigalsidase alfa-iwxj
Dosage form: Injection
Company: Protalix BioTherapeutics, Inc.
Treatment for: Fabry Disease
Elfabrio (pegunigalsidase alfa-iwxj) is a hydrolytic lysosomal neutral glycosphingolipid-specific enzyme indicated for the treatment of adults with confirmed Fabry disease.
- Fabry disease is an X–linked inherited neurological disorder caused by a deficiency of the alpha-galactosidase-A enzyme, resulting in progressive accumulation of abnormal deposits of a fatty substance called globotriaosylceramide (Gb3) in the lysosomes throughout a person's body. Symptoms range from episodes of pain and impaired peripheral sensation to end-organ failure.
- Elfabrio is an enzyme replacement therapy that works to treat Fabry disease by providing an exogenous source of alpha-galactosidase A.
- Elfabrio is administered as an intravenous infusion every two weeks. Pretreatment with antihistamines, antipyretics, and/or corticosteroids may be considered.
- The Elfabrio product label carries a Boxed Warning for hypersensitivity reactions, including anaphylaxis. Warnings and precautions include infusion-associated reactions and membranoproliferative glomerulonephritis.
- Common adverse reactions include infusion-associated reactions, nasopharyngitis, headache, diarrhea, fatigue, nausea, back pain, pain in extremity, and sinusitis.
Development timeline for Elfabrio
Further information
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