idebenone (Raxone)

following a full submission assessed under the ultra-orphan medicine process:

idebenone (Raxone®) is accepted for restricted use within NHS Scotland.

Indication under review: Treatment of visual impairment in adolescent and adult patients with Leber’s Hereditary Optic Neuropathy (LHON).  

SMC restriction: to patients with LHON who are not yet blind i.e. they do not meet the UK criteria to be registered as severely sight impaired.

In a 24-week double-masked randomised placebo-controlled study, patients who received idebenone had numerical improvements in visual acuity over placebo.
 
This SMC advice takes account of the benefits of a Patient Access Scheme (PAS) that improves the cost-effectiveness of idebenone. This advice is contingent upon the continuing availability of the PAS in NHS Scotland or a list price that is equivalent or lower.

This advice takes account of views from a Patient and Clinician Engagement (PACE) meeting.