- Cancer Biology & Biomarkers
- Chromatography & Mass Spectrometry
- Contract Research, Clinical Trials and Outsourcing
- Drug Discovery
- Drug Targets
- Flow Cytometry
- Informatics & Lab Automation
- Ingredients, Excipients and Dosages
- Microbiology & RMMs
- NIR, PAT & QbD
- Raman Spectroscopy
- Screening, Assays & High-Content Analysis
- Thermal Processing
- Events & Workshops
Santhera launches Raxone in its first EU market
2 October 2015 • Author: Victoria White
Santhera Pharmaceuticals has launched Raxone for the treatment of Leber’s Hereditary Optic Neuropathy (LHON) in Germany, its first and largest EU market.
LHON is a rare inherited mitochondrial disease that usually leads rapidly to profound and permanent blindness. The disease presents predominantly in young, otherwise healthy adult males as rapid, painless loss of central vision, usually leading to permanent bilateral blindness within a few months of the onset of symptoms. About 95% of patients harbor one of three pathogenic mutations of the mitochondrial DNA, which cause a defect in the complex I subunit of the mitochondrial respiratory chain. This defect leads to decreased cellular energy (ATP) production, increased reactive oxygen species (ROS) production and retinal ganglion cell dysfunction, which cause progressive loss of visual acuity and blindness.
Raxone circumvents the complex I defect, reduces and scavenges ROS, restores cellular energy levels in retinal ganglion cells and promotes recovery of visual acuity. Efficacy data come from Santhera’s randomised, placebo-controlled RHODOS trial and from the open label Expanded Access Programme, which together have demonstrated that vision loss can be mitigated or reversed in patients treated with Raxone.
Raxone is the first approved treatment for any mitochondrial disease
“The introduction of Raxone in Germany is a major step forward for LHON patients who now have an effective treatment option” said Giovanni Stropoli, Santhera’s Chief Commercial Officer for Europe. “We are particularly proud as Raxone is also the first approved treatment for any mitochondrial disease, a therapeutic area which is in the focus of our Company. Germany is our first and largest EU market but product availability is anticipated in additional countries in the immediate future.”
“Mitochondrial disease treatments have been elusive for a long time. The availability of Raxone for patients with LHON is changing the paradigm and also raises hopes for other mitochondrial disorders,” said Thomas Klopstock, MD, Professor of Neurology at the University of Munich, LHON researcher and coordinator of the German network for mitochondrial disorders, mitoNET. “LHON patients usually become blind in young adulthood, which entails not only a personal and medical, but also a socioeconomic burden. With Raxone, we can now offer a treatment option which greatly improves the chances for significant recovery of visual acuity.”
ABB Analytical Measurement ACD/Labs ADInstruments Ltd Advanced Analytical Technologies GmbH Analytik Jena AG Astell Scientific Ltd B&W Tek Bachem AG Bibby Scientific Limited Bio-Rad Laboratories BioNavis Ltd Biopharma Group Black Swan Analysis Limited Charles Ischi AG | Kraemer Elektronik Cherwell Laboratories CI Precision Cobalt Light Systems Coulter Partners CPC Biotech srl Dassault Systèmes BIOVIA DiscoverX Edinburgh Instruments Enterprise System Partners (ESP) EUROGENTEC F.P.S. Food and Pharma Systems Srl IDBS JEOL Europe L.B. Bohle Maschinen + Verfahren GmbH Lab M Ltd. LabWare Linkam Scientific Instruments Limited Molins Technologies Multicore Dynamics Ltd Nanosurf New England Biolabs, Inc. Panasonic Biomedical Sales Europe B.V. PerkinElmer Inc ReAgent Russell Finex Limited Source BioScience Takara Clontech Tornado Spectral Systems Tuttnauer Watson-Marlow Fluid Technology Group Wickham Laboratories Limited Xylem Analytics YMC Europe GmbH Yusen Logistics