Idebenone

How it works

It is thought to act as an antioxidant and electron carrier, supporting mitochondrial electron transport and ATP production while reducing oxidative stress in affected retinal ganglion cells.

Prescribing in practice

• Discontinue if there is no evidence of benefit after an adequate treatment period, and review response according to the SPC, as not all patients recover vision.
• It may discolour urine, which is harmless but worth flagging to avoid alarm.
• Use cautiously in hepatic or renal impairment and review concomitant medication as advised in current prescribing references.

Monitoring

Monitor visual acuity periodically to assess response and inform decisions on continuing therapy.

Counselling the patient

• Explain that any visual recovery is typically gradual and not guaranteed.
• Take the medicine with food as directed.
• A reddish-brown discolouration of urine is expected and not harmful.

Evidence & guidelines

Idebenone is approved for Leber's hereditary optic neuropathy on the basis of trials demonstrating recovery of visual acuity in a proportion of treated patients.