Nature Communications Publishes Promising Results for Idorsia’s Lucerastat Improving Kidney Function in Fabry Disease

Idorsia Pharmaceuticals Ltd has announced the publication of pivotal data on its investigational oral therapy lucerastat in Nature Communications, highlighting its promising impact on kidney function in adults living with Fabry disease. The findings come from the Phase 3 MODIFY study and its open‑label extension (OLE), which evaluated lucerastat as a substrate reduction therapy in a diverse cohort of 118 patients across 14 countries. While lucerastat did not meet the primary endpoint of reducing neuropathic pain during the double‑blind phase, it showed robust reductions in key biomarkers (plasma and urinary Gb3) compared with placebo, sustained over time in the extension study. Most notably, an interim analysis of patients treated for at least 12 months revealed a slower decline in eGFR, especially in those with impaired or rapidly deteriorating kidney function at baseline—suggesting a potential disease‑modifying effect on renal decline. Long‑term data (up to 42 months and beyond 6 years for some) confirmed lucerastat’s tolerability and safety, with no treatment‑related serious adverse events. A kidney biopsy substudy further supports continued investigation. Idorsia is collaborating with regulatory authorities, including the US FDA, to determine the optimal pathway for approval and is establishing a post‑trial access program for ongoing care after the OLE ends. #FabryDisease #Lucerastat #Idorsia #KidneyFunction #SlimScan #GrowthStocks #CANSLIM