A pivotal Phase 3 trial reported encouraging results for lucerastat, an oral therapy for Fabry disease, showing meaningful clinical signals versus placebo in key endpoints. Trial investigators and company data indicate improvements in symptom measures and biomarkers consistent with disease modification. If validated in full analysis and regulatory submissions, an oral small‑molecule treatment would expand options for Fabry patients who now rely largely on enzyme replacement or other modalities. Sponsors are preparing regulatory strategies and potential labeling discussions based on the pivotal dataset.