Santhera Pharmaceuticals Holding AG : Santhera Receives a Negative Opinion on its Marketing Authorization Application for

Santhera Pharmaceuticals Holding AG : Santhera Receives a Negative Opinion on
    its Marketing Authorization Application for Raxone®. Plans to Request
                                Re-examination

Santhera Pharmaceuticals Holding AG / Santhera Receives a Negative Opinion on
its Marketing Authorization Application for Raxone®. Plans to Request
Re-examination . Processed and transmitted by Thomson Reuters ONE. The issuer
is solely responsible for the content of this announcement.



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.

                                    * * *

About Santhera
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
thomas.meier@santhera.com

Disclaimer / Forward-looking statements

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.

News release CHMP

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Hammerstrasse 49 Liestal Switzerland

ISIN: CH0027148649;
 
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