Raxone has demonstrated its efficacy and safety profile in LHON in the largest clinical development program ever undertaken in LHON.1–7
The clinical development program included a double-blind, randomized, placebo-controlled study (RHODOS), an observational follow-up study (RHODOS-OFU), an Expanded Access Program (EAP), and a natural history case record survey (CRS).1,3–5,8–12 A number of additional clinical studies in multiple indications also support the safety profile of Raxone.9–12
RHODOS is the first and only randomized, placebo-controlled clinical trial to be completed in LHON.3,4
An observational follow-up study (RHODOS-OFU) was conducted in 58 patients who completed the RHODOS study and who were assessed after 30 months (median) off-treatment in order to assess whether treatment effects persisted over time.4
Raxone Expanded Access Program (EAP)
Natural History Case Record Survey (CRS)
Raxone SmPC, September 2015. Available here. Accessed December 2015.
EMA. EPAR summary for the public: Raxone (idebenone). 2015
Klopstock T, et al. Brain 2011; 134:2677–2686
Klopstock T, et al. Brain 2013; 136:e230
Metz G, et al. ARVO 2014 poster (Abstract 6206)
Gueven N & Faldu D. Expert Opin Orphan Drugs 2013; 1:331–339
Rudolph G, et al. J Neuroophthalmol 2013; 33:30–36
Metz G, et al. EVER 2014 poster (Abstract 2658)
Di Prospero NA, et al. Arch Neurol 2007; 64:803–808.
Lynch DR, et al. Arch Neurol 2010; 67:941–947.
Buyse GM, et al. Lancet 2015; 385:1748–1757.
Buyse GM, et al. Neuromuscul Disord 2011; 21:396–405.
EMA. Assessment report: Raxone (idebenone). 2015.