Health Care Professionals


Santhera is passionate about developing new treatments for patients with mitochondrial diseases. Our focus is on the development of treatments for neuromuscular and neuro-ophthalmological diseases that currently lack treatment options.

Raxone in LHON: Clinical development

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

The RHODOS trial

RHODOS is the first and only randomized, placebo-controlled clinical trial to be completed in LHON.3,4

Figure created using information from: Klopstock T, et al. Brain 2011; 134:2677–2686 and Klopstock T, et al. Brain 2013; 136:e230.

What additional studies have been performed?

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)

  • The EAP provides insight into Raxone’s therapeutic potential in a real-world setting.5
  • Raxone was provided under the Named Patient Program (EU) or Treatment Investigational New Drugs (US) regulations.5
  • Enrollment was limited to patients presenting for treatment within 1 year of onset of symptoms.5
  • Data from 69 patients at 36 centers worldwide were analysed.
  • There will be no new patients enrolled in the EAP

Natural History Case Record Survey (CRS)

  • The CRS was established as a collaboration between Santhera Pharmaceuticals and the European Vision Institute Clinical Research Network (
  • Historically documented visual acuity (VA) data from existing medical records were collected from participating member centers and LHON-treating centers in the EAP across Europe and the US.13



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.

This section of the website is intended
to provide healthcare professionals with
information about Raxone® and other
products in Santhera’s pipeline.

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