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.
Download detailed advice355KB (PDF)
- Medicine name:
- idebenone (Raxone)
- SMC ID:
- Treatment of visual impairment in adolescent and adult patients with Leber' s Hereditary Optic Neuropathy (LHON).
- Pharmaceutical company
- Santhera Pharmaceuticals
- BNF chapter
- Submission type
- Date advice published
- 08 May 2017