Nomlabofusp shows potential in treating Friedreich's ataxia, with 100% of participants achieving normal skin FXN levels.

A 6-month open-label study found that 100% of participants achieved skin FXN levels similar to asymptomatic carriers after daily nomlabofusp administration. Consistent directional improvement was seen across multiple measures after 1-year, suggesting nomlabofusp may alter the disease course of Friedreich's ataxia. Anaphylaxis was experienced by 7 participants in the first 6 weeks of dosing, but long-term dosing was generally well-tolerated.