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2014-05-07

Santhera Pharmaceuticals Holding AG: Santhera Files Marketing Authorization Application in European Union for Raxone® in the Treatment of LHON

Santhera Pharmaceuticals Holding AG / Santhera Files Marketing Authorization
Application in European Union for Raxone®in the Treatment of LHON . Processed
and transmitted by NASDAQ OMX Corporate Solutions.The issuer is solely
responsible for the content of this announcement.
Liestal, Switzerland, May 7, 2014
- Santhera Pharmaceuticals (SIX: SANN) announced today
that it has re-filed
with the
European Medicines Agency (EMA) a Marketing Authorization Application (MAA)
for
Raxone®

(
idebenone
)
in the treatment of Leber
'
s Hereditary Optic Neuropathy (LHON). An earlier application was withdrawn in
March 2013 in order to include additional clinical efficacy data.
The compound has
been granted orphan drug designation in the European Union (EU).
Raxone®
would become the first product authorized for the treatment of
this rare, inherited disease which otherwise invariably leads to blindness.
Santhera expects a decision from the EMA in the first half of 2015.

Santhera discussed the additional clinical efficacy data and the overall
content of the revised MAA dossier with several EU member states prior to
proceeding with the re-filing. Earlier this year the French National Agency
for the Safety of Medicine and Health Products (ANSM) granted a temporary
authorization for use for Raxone® in LHON patients in France based on a data
package comparable to the submitted MAA dossier.

"We are optimistic that the new data on Raxone® which we have submitted in
this MAA dossier will address the issues raised by the CHMP during the
previous procedure" commented Thomas Meier, Chief Executive Officer of
Santhera. "The totality of data included in the re-submission provides
compelling evidence that Raxone® can protect against vision loss in the early
stages of the disease and promote recovery of vision already lost."

Clinical data presented at ARVO conference in the US

Efficacy data on Raxone® in LHON patients from an ongoing Expanded Access
Program will be presented this week at the Annual Meeting of the Association
for Research in Vision and Ophthalmology (ARVO) in Orlando, Florida. The
poster can be downloaded
fromwww.santhera.comunderProducts&Pipeline/Publications
.

US regulatory strategy
Santhera has previously been granted an US orphan drug designation for the
treatment of LHON and will now approach the US Food and Drug Administration
(FDA) for discussions on a regulatory path to an US approval based on the
data package filed in the European MAA.

About LHON and the treatment with Raxone
®
Leber's Hereditary Optic Neuropathy (LHON) is a heritable genetic disease
which usually leads to permanent blindness. The disease typically presents in
young adult men as rapid, painless loss of central vision in one eye,
followed by the fellow eye within a few weeks or months of the onset of
symptoms. Over 90% of patients harbour one of three pathogenic mutations of
their 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 to retinal ganglion
cell dysfunction which cause progressive loss of visual acuity and blindness.

Raxone® (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 bypassing the complex I defect, restoring ATP production in
retinal ganglion cells and promoting recovery of visual acuity.

* * *

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 mitochondrial and
neuromuscular diseases, such as Leber's Hereditary Optic Neuropathy, Duchenne
Muscular Dystrophy and primary progressive Multiple Sclerosis, all of them
areas of high unmet medical need with no current therapies. For further
information, please visit the Company's websitewww.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.

News release MAA
http://hugin.info/137261/R/1783267/610642.pdf

---------------------------------------

This announcement is distributed by NASDAQ OMX Corporate Solutions on behalf of NASDAQ OMX Corporate Solutions clients.
The issuer of this announcement warrants that they are solely responsible for the content, accuracy and originality of the information contained therein.
Source: Santhera Pharmaceuticals Holding AG via Globenewswire

HUG#1783267

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