9 May 2017 - Santhera Pharmaceuticals announces approval by the Scottish Medicines Consortium for restricted use of Raxone (idebenone) in patients with LHON, with effect May 8, 2017.

Patients with Leber’s hereditary optic neuropathy (LHON), an extremely rare, inherited cause of blind- ness, could benefit from the only licensed treatment to date, following advice issued by the Scottish Medicines Consortium (SMC) under the ultra-orphan medicine process. The SMC accepted Raxone (idebenone) for restricted use by NHS Scotland in the treatment of visual impairment in adolescent and adult patients with LHON who are not yet blind (i.e., they do not meet the UK criteria to be registered as severely sight impaired). 

Raxone was accepted following consideration through SMC’s Patient and Clinician Engagement (PACE) process, for medicines used at the end of life and for very rare conditions. Scotland is the first country in the UK to make Raxone available for the treatment of LHON. The SMC advice takes into account the benefits of a Patient Access Scheme, which improves the cost-effectiveness of Raxone.

Read Santhera Pharmaceuticals press release