Health Care Professionals


Santhera is passionate about medical science and about developing new medical treatments for patients with rare diseases. Our focus is on the development of treatments for neuromuscular and pulmonary 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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