Summary

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• Elfabrio (pegunigalsidase alfa-iwxj) is indicated for the treatment of adults with confirmed Fabry disease.
• This summary is based on the review of one randomized controlled trial(s). ^[1]
• Renal Function and Gb3 Reduction in Classic Fabry Disease (FD): Treatment with pegunigalsidase alfa for one year resulted in an 84% reduction in renal peritubular capillary globotriaosylceramide (Gb3) inclusions in patients with the classic FD phenotype. The mean estimated glomerular filtration rate (eGFR) remained stable at 111 mL/min/1.73 m² from baseline throughout the treatment period.
• Pharmacokinetics: Pegunigalsidase alfa exhibited a mean terminal plasma half-life ranging from 53 to 121 hours, indicating prolonged circulation time.
• Cardiac and Clinical Parameters: The study mentions that cardiac parameters and other clinical endpoints were assessed, but specific results are not provided.
• Nearly all treatment-emergent adverse events (AEs) were mild or moderate in severity; one patient withdrew from the study due to a serious related adverse event.
• Three patients developed treatment-induced immunoglobulin G (IgG) antidrug antibodies (ADAs), all of whom became ADA-negative after one year of treatment. 8. duced an 84% reduction in renal peritubular capillary Gb3 inclusions in patients with the classic Fabry disease (FD) phenotype, with 11 males and 1 of 7 females presenting this phenotype; immunogenicity was noted in three patients who developed IgG antidrug antibodies, but all became ADA-negative after one year of treatment.