Drug updated on 4/17/2024
| 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.
Summary
- Pegunigalsidase alfa-iwxj (Elfabrio) is indicated for the treatment of adults with confirmed Fabry disease, a genetic disorder. The medicine aims to increase plasma half-life and reduce immunogenicity to enhance efficacy over existing treatments.
- One randomized controlled study focused on the safety, pharmacokinetics, and immunogenicity of Elfabrio as an enzyme replacement therapy for Fabry disease.
- The study involved sixteen symptomatic adults with Fabry disease, including 11 males and 5 females. Pegunigalsidase alfa demonstrated a significant reduction in renal peritubular capillary globotriaosylceramide (Gb3), a pathological marker associated with the classic phenotype of Fabry's Disease.
- The terminal plasma half-life ranged from 53 to 121 hours across different cohorts receiving increasing doses via intravenous infusion every other week. This suggests that Elfabrio has improved pharmacokinetic properties compared to other available treatments.
- Three patients developed IgG antidrug antibodies during treatment, but all became antibody-negative after one year, indicating potential transient immunogenicity. Most adverse events were mild or moderate, suggesting a favorable safety profile for Elfabrio in treating adult patients diagnosed with FD.
- Although direct comparisons between pegunigalsidase alfa and other medications are not provided within this study, it can be inferred that its unique design aimed at improving pharmacokinetics while reducing immunogenicity may offer advantages over existing therapies used in managing FD among adult patient populations.
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