Santhera Receives a Negative Opinion on its Marketing Authorization Application for Raxone®. Plans to Request Re-examination
Liestal, Switzerland, January 18, 2013 - Santhera Pharmaceuticals (SIX: SANN) announced today that it has received a negative opinion on its Marketing Authorization Application (MAA) for Raxone® as a potential therapy for Leber's Hereditary Optic Neuropathy (LHON). The European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) has notified Santhera that a narrow majority of CHMP members deemed Raxone® not approvable at this time. Santhera believes that the clinical benefit of Raxone® in patients in whom the medical need for treatment was most urgent had not been fully considered and therefore has decided to request a re-examination of the opinion.
Thomas Meier, Chief Executive Officer of Santhera, commented, "We are disappointed that the CHMP did not follow the recommendation for approval proposed by the Rapporteurs. In the Rapporteurs' view sufficient data had been provided in this very rare disease demonstrating a consistent and significant clinical benefit in LHON patients in whom the medical need is most urgent and the probability of benefit the highest. These were exactly the patients we targeted in this application. However, a narrow majority of CHMP delegates voiced concerns about the reliability of the results due to the small number of patients studied in this category and voted against approval at this time. Since there were no concerns expressed about the safety of Raxone® and the observed treatment benefit on visual acuity in these patients was clinically relevant and consistent with the published literature, we believe that we should be allowed to address the remaining concerns through further confirmatory clinical work to be conducted post-approval. To this extent, we continue to strive for approval so that Raxone® becomes available as the first treatment for patients with this devastating disease".
The outcome on the re-examination can be expected to be known by mid 2013.
About Raxone® in LHON
Santhera develops Raxone® as treatment for patients with LHON, a heritable genetic disease causing blindness. LHON typically presents in young adults, mostly men, as painless loss of vision in both eyes, leading to blindness within a few months of the onset of symptoms. Over 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 oxidative stress and retinal ganglion dysfunction which cause progressive loss of visual acuity and blindness.
Idebenone, a synthetic short-chain benzoquinone and a cofactor for the enzyme NAD(P)H:quinone oxidoreductase (NQO1) is capable of transferring electrons directly onto complex III of the mitochondrial electron transport chain, thereby circumventing the complex I defect and restoring cellular energy levels. By this mechanism of bypassing complex I, which is affected in all three primary mitochondrial DNA mutations causing LHON, idebenone supports electron transport and cellular energy generation in retinal ganglion cells, thereby promoting recovery of visual acuity.
The efficacy of Raxone® has been tested in a randomized, placebo controlled study and confirmed in a number of open label cohort studies and case reports by independent academic experts.
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Santhera Pharmaceuticals (SIX: SANN) is a Swiss specialty pharmaceutical company focused on the development and commercialization of innovative pharmaceutical products for the treatment of orphan neuromuscular and mitochondrial diseases, areas of high unmet medical need with no current therapies. For further information, please visit www.santhera.com.
Raxone® is a trademark of Santhera Pharmaceuticals.
For further information, please contact
Thomas Meier, Chief Executive Officer
Phone: +41 61 906 89 64
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This communication does not constitute an offer or invitation to subscribe for or purchase any securities of Santhera Pharmaceuticals Holding AG. This publication may contain certain forward-looking statements concerning the Company and its business. Such statements involve certain risks, uncertainties and other factors which could cause the actual results, financial condition, performance or achievements of the Company to be materially different from those expressed or implied by such statements. Readers should therefore not place undue reliance on these statements, particularly not in connection with any contract or investment decision. The Company disclaims any obligation to update these forward-looking statements.