A pivotal Phase 3 trial begins for an oral therapy aimed at Fabry disease's kidney damage

Fabry disease is a rare inherited condition in which a fatty substance called Gb3 quietly builds up in cells, damaging the kidneys, heart and nervous system over years. Switzerland's Idorsia has now started FAB-KLEAR, a pivotal Phase 3 study of lucerastat — an oral therapy designed to slow that build-up at its source, whatever a patient's exact genetic mutation or previous treatment. Japan's Nxera Pharma has exercised its option to co-develop the drug and will run development in Japan and selected Asia-Pacific markets, paying CHF 7 million (about $8.7 million) upfront and sharing the global trial costs. Lucerastat already has more than 400 patient-years of clinical experience behind it, including patients treated for over six years. 'We are entering an important new phase,' said Idorsia chief executive Roland Wandeler — one that could end in a regulatory filing as early as 2029.

SDG 3 · Good health and well-being — Mechanism: tech innovation