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NICE Approves Idebenone for LHON on NHS in England

About 250 patients over 12 in England are now eligible, marking NICE’s first approval for a mitochondrial disease.

Overview

  • The treatment targets energy production in affected eye cells, with studies indicating improvements in vision and quality of life.
  • Eligibility covers people aged 12 and above with LHON, and evidence shows roughly half of treated patients see benefit.
  • The medicine, branded Raxone by Chiesi, is taken as two tablets three times a day.
  • The decision brings England into line with Wales and Scotland, where access had been available on a limited basis.
  • LHON is a rare inherited disorder that can cause rapid, severe vision loss and affects an estimated 2,000 people in England.