Summary

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• This summary is based on the review of one randomized controlled trial(s). ^[1]
• Pegunigalsidase alfa demonstrated non-inferiority to agalsidase beta in the primary efficacy outcome of annualized eGFR slope over 2 years. The difference in median eGFR slopes between the two groups was -0.36 mL/min/1.73 m²/year, meeting the prespecified non-inferiority margin.
• Minimal changes were observed in lyso-Gb3 concentrations in both treatment arms at the end of the 2-year period, further supporting the non-inferiority of pegunigalsidase alfa compared to agalsidase beta.
• Subgroup analysis based on baseline characteristics such as age and gender (mean age of 44 years, 61% male) did not reveal significant differences in effectiveness outcomes among these subgroups.
• The safety profile of pegunigalsidase alfa was favorable compared to agalsidase beta, with exposure-adjusted rates of treatment-related adverse events being 3.6 times lower and mild or moderate infusion-related reactions being 7.8 times lower for pegunigalsidase alfa. Neutralizing antibodies were detected in 15% of pegunigalsidase alfa-treated patients compared to 26% of those treated with agalsidase beta.
• There were no deaths reported during the study, and no specific safety concerns or adverse effects were noted in particular population types or subgroups.
• The study population consisted of adults with Fabry disease who had an annualized eGFR slope more negative than -2 mL/min/1.73 m²/year and had received agalsidase beta for at least one year. The mean age of participants was 44 years, with 61% being male. No significant differences in effectiveness or safety outcomes were noted between subgroups based on age or gender in the abstract.