Pegunigalsidase alfa, marketed as Elfabrio, is a therapeutic enzyme used as long-term enzyme replacement therapy for adults with confirmed Fabry disease, a rare genetic disorder involving deficiency of the enzyme alpha-galactosidase. It is a recombinant human alpha-galactosidase-A, a hydrolytic lysosomal neutral glycosphingolipid-specific enzyme, and it is administered intravenously. Common adverse reactions include infusion-related reactions, hypersensitivity responses and asthenia. It received approval in both the European Union and the United States in May 2023, after the European Medicines Agency's Committee for Medicinal Products for Human Use issued a positive recommendation in February 2023. The applicant company was Chiesi Farmaceutici.
Facts
Classification
Approval Status ATC ClassSourced to the subject's own accountA: Alimentary Tract and Metabolism 2 Route of Administration First Approved / Isolated Year Sources
1. Pegunigalsidase alfa (Wikipedia)
Lead section, approval-status statementQuote, Lead section, approval-status statement
Pegunigalsidase alfa was approved for medical use in both the European Union and the United States in May 2023.
2. Anatomical Therapeutic Chemical Classification System (Wikipedia)
WikipediaFirst-level anatomical group codes, Code AQuote, First-level anatomical group codes, Code A
Alimentary tract and metabolism
3. Pegunigalsidase alfa (Wikipedia)
WikipediaInfobox drug, routes_of_administrationQuote, Infobox drug, routes_of_administration
Intravenous
4. Drugs.com: Elfabrio (pegunigalsidase alfa-iwxj) FDA Approval History
FDA Approval HistoryQuote, FDA Approval History
Elfabrio (pegunigalsidase alfa-iwxj) was first approved by the FDA on May 9, 2023
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