Drug updated on 9/4/2024
Dosage Form | Injection (intravenous; 20 mg/10 mL [2 mg/mL] solution in a single-dose vial) |
Drug Class | Hydrolytic lysosomal enzymes |
Ongoing and Completed Studies | ClinicalTrials.gov |
Indication
- Indicated for the treatment of adults with confirmed Fabry disease.
Latest News
Summary
- 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.
Product Monograph / Prescribing Information
Document Title | Year | Source |
---|---|---|
Elfabrio (pegunigalsidase alfa-iwxj) Prescribing Information. | 2023 | Chiesi Farmaceutici S.p.A., Parma, Italy |
Randomized Controlled Trials
Document Title | Sex Distribution | Year | Source |
---|---|---|---|
Pegunigalsidase alfa, a novel PEGylated enzyme replacement therapy for Fabry disease, provides sustained plasma concentrations and favorable pharmacodynamics: A 1-year Phase 1/2 clinical trial. | 18Subjects F: 39% M: 61% | 2019 | Journal of Inherited Metabolic Disease |
Sex Distribution:
F:39%
M:61%
18Subjects
Year:
2019
Source:Journal of Inherited Metabolic Disease