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: Mode of Action

Raxone facilitates mitochondrial electron flux by bypassing the deficient complex I of the mitochondrial respiratory chain in LHON, and so restores cellular adenosine triphosphate (ATP) generation.1–5 As a result, it may reactivate viable-but-inactive retinal ganglion cells (RGCs).4,6,7 This, in turn, can promote recovery of vision in patients with LHON who experience vision loss.

Raxone also has potent antioxidant capacity, demonstrated by its ability to prevent lipid peroxidation and to protect against reactive oxygen species (ROS)-induced damage.5,8

An early diagnosis of LHON offers patients the best chance for the treatment of their condition in the long term.9 There is a window of opportunity for the optimal treatment of LHON when RGCs are still viable, meaning Raxone should be initiated as soon as possible to maximize treatment response.10,11

For an overview of Raxone´s Mode of Action in LHON, please click on the video below.

To download the Raxone Mode of Action video, please click here 



Gueven N & Faldu D. Expert Opin Orphan Drugs 2013; 1:331–339.


Gueven N et al. Redox Biol 2015; 4:289–295.


EMA. EPAR summary for the public: Raxone (idebenone). 2015.


Raxone SmPC, September 2015. Accessed December 2015. Available here


Haefeli RH, et al. PLoS One 2011; 6:e17963.


Howell N. Vision Res 1998; 38:1495–1504.


Heitz FD, et al. PLoS One 2012; 7:e45182.


Erb M, et al. PLoS One 2012; 7:e36153.


Sadun AA, et al. Expert Rev Ophthalmol 2012; 7:251–259.


Gueven N. Biol Med 2014; 6(S3):1–6.


Newman NJ. Nat Rev Neurol 2012; 8:545–556.

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

I am a Healthcare Professional
I am not a Healthcare Professional