Idebenone
(Brand name Raxone)
Approved in Europe for adults and adolescents aged 12 years and over with vision loss from LHON. The Therapeutic Goods Administration (TGA) is currently considering whether Raxone should be approved for LHON in Australia.
Which people with mito might this therapy help?Â
People with Leber hereditary optic neuropathy (LHON), especially people who have had recent vision loss.
Idebenone is designed to help mitochondria make energy. In LHON, it may help cells in the optic nerve that are still alive but not working well.
People should speak with their eye specialist, neurologist or mitochondrial specialist about whether this treatment may be suitable for them.
What research has been done on this therapy?Â
Idebenone has been studied in people with LHON.
The RHODOS study was a Phase 2/3 trial. It included people aged 14 to 65 years who had vision loss from LHON and one of the three main LHON mitochondrial DNA variants: m.11778G>A, m.14484T>C or m.3460G>A.
The LEROS study was a Phase 4 study. It looked at long-term Raxone treatment in people with LHON. The study included 199 people and is now completed.
Important information
Find out more
Emerging therapies for mito
Learn about developing therapies and opportunities to take part in research.
Clinical trials explained
A guide to help understand participation and make informed decisions.
