Editor’s note: This is a developing news story. Please check back soon for updates.
The FDA granted priority review to idebenone for the treatment of Leber hereditary optic neuropathy, according to a press release from Chiesi Global Rare Diseases.
Idebenone, a short-chain benzoquinone designed to target mitochondrial dysfunction in retinal ganglion cells, was assigned a Prescription Drug User Fee Act (PDUFA) date of Feb. 28, 2026. The submission is supported by positive results from the phase 3 RHODOS and phase 4 LEROS studies.
The RHODOS study, which included 85 patients (Read more...)