Santhera Pharmaceuticals Holding AG : Santhera Withdraws Marketing Authorization Application for Raxone® in LHON

      Santhera Pharmaceuticals Holding AG : Santhera Withdraws Marketing
                Authorization Application for Raxone® in LHON

Santhera Pharmaceuticals Holding AG / Santhera Withdraws Marketing
Authorization Application for Raxone® in LHON . Processed and transmitted by
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Liestal, Switzerland, March22, 2013 - Santhera Pharmaceuticals (SIX: SANN)
announced today its decision to withdraw, for strategic reasons, the Marketing
Authorization Application (MAA) for Raxone^® as a potential therapy for
Leber's Hereditary Optic Neuropathy (LHON). Santhera now plans to file a new
application based on emerging clinical evidence supporting the efficacy of
Raxone^® in the treatment of LHON. This strategy is expected to increase the
probability of a successful MAA outcome and is contingent on the availability
of sufficient financial resources or on the outcome of ongoing licensing and
M&A discussions.

Thomas Meier, Chief Executive Officer of Santhera, commented: "We initially
planned to request re-examination of the previous CHMP opinion on our MAA in
LHON. However, in view of emerging clinical evidence of efficacy, which the
EMA confirmed cannot be considered in a re-examination procedure, we have
decided to withdraw the MAA and to initiate a new procedure including this new
evidence. Supported by external expert clinical and regulatory advice, we
believe that this will allow us to address adequately the points raised by the
CHMP in their opinion and increase the probability of an ultimately successful
outcome."

As indicated in late February 2013, Santhera continues to explore strategic
and financing options including product licensing for Raxone^® in LHON and the
possibility of a merger or acquisition. In addition, Santhera anticipates
convening a Shareholders' meeting in late April/early May 2013 to present the
strategic options available and to allow a decision to be reached on the
future direction of the Company.

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 cell 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 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, 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.

Withdrawal

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Santhera Pharmaceuticals Holding AG
Hammerstrasse 49 Liestal Switzerland

ISIN: CH0027148649;
 
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