A study on lucerastat shows a potential disease-modifying effect in Fabry disease patients with impaired renal function or fast-deteriorating eGFR. The Phase 3 MODIFY study and open-label extension demonstrated a reduction in kidney function loss compared to placebo. While the study did not meet its primary endpoint of reducing neuropathic pain, lucerastat significantly reduced plasma and urinary Gb3 levels. Ongoing patients treated with lucerastat for at least 12 months showed improved renal function. The company is working with health authorities to seek approval for this promising therapy. Fabry disease is a rare lysosomal storage disorder affecting multiple organ systems and has a high prevalence worldwide.

Read more at GlobeNewswire: Nature Communications reports promising effect of Idorsia’s