Head-to-head trial of pegunigalsidase alfa versus agalsidase beta in patients with Fabry disease and deteriorating renal function: results from the 2-year randomized phase 3 BALANCE study

BALANCE is the first study directly comparing pegunigalsidase alfa with agalsidase beta for the treatment of patients with Fabry Disease (FD). At enrollment, participants had received previous agalsidase beta treatment for 6 years on average and had worsening kidney function. The participants were then randomly assigned to receive pegunigalsidase alfa or continue agalsidase beta. Results show these enzyme replacement therapies were comparable in how they affected the rate at which kidney function declined following random assignment. Participants tolerated pegunigalsidase alfa well and had lower rates of adverse events, especially infusion-related reactions, compared with participants receiving agalsidase beta. Pegunigalsidase alfa offers a new treatment option for adults with FD who have worsening kidney function. (By Drs David G Warnock and Eric L Wallace, https://jmg.bmj.com/content/early/2023/11/07/jmg-2023-109445 )

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