Minoryx is on the cusp of a significant breakthrough in treating cerebral adrenoleukodystrophy (cALD) with its drug, leriglitazone. The European Medicines Agency’s Committee for Medicinal Products for Human Use has recommended approval for boys aged 2 to 12 with early-stage, non-gadolinium-enhancing brain lesions and minimal neurological impairment, following a new application supported by the NEXUS study. Although the broader patient population targeted was narrowed due to earlier trial failures, the new findings showed that 35% of treated boys experienced disease stabilization. The European Commission is expected to make the final decision soon, while Minoryx continues additional trials for other conditions.
Disclaimer: This summary is written by AI, which can make mistakes. Please follow up on any news information from additional sources.
Link to original article source: https://european-biotechnology.com/latest-news/chmp-backs-minoryxs-rare-disease-drug-two-years-after-rejection/
