2013-01-18 07:05:10 -
Santhera Pharmaceuticals Holding AG /
Santhera Receives a Negative Opinion on its Marketing Authorization Application
for Raxone®. Plans to Request Re-examination
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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
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.
* * *
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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