CHMP recommends granting a marketing authorization for Santhera's Raxone® for the treatment of Leber's Hereditary Optic Neuropathy (LHON)
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Santhera Pharmaceuticals Holding AG /
CHMP recommends granting a marketing authorization for Santhera's Raxone® for
the treatment of Leber's Hereditary Optic Neuropathy (LHON)
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The issuer is solely responsible for the content of this announcement.
Liestal, Switzerland, June 26, 2015 - Santhera Pharmaceuticals (SIX: SANN)
announces that the European Medicines Agency's Committee for Medicinal Products
for Human Use (CHMP) has recommended granting a marketing authorization for
Raxone(®) (idebenone) for the treatment of visual impairment in adolescent and
adult patients with LHON.
LHON is a heritable mitochondrial disease that leads to rapid, profound and
usually permanent blindness in otherwise healthy patients. Raxone(®) will be the
first treatment option for LHON and the first approved therapy for a
mitochondrial disease.
"This is a major breakthrough as it paves the way for the first medicinal
product to become available for the treatment of a mitochondrial disease,"
stated Thomas Klopstock, MD (Professor for Neurology at the University of
Munich, LHON investigator and coordinator of the German network for
mitochondrial disorders, mitoNET). "LHON is a severe form of vision loss caused
by mitochondrial dysfunction. Affected patients, usually young and otherwise
healthy, rapidly lose central vision and become bilaterally blind within a few
months from the onset of symptoms. Although there is a chance for partial or
even full spontaneous recovery, most patients remain permanently blind if
untreated. The mode of action of idebenone provides a clear biochemical and
medical rationale and the clinical data demonstrate that vision of affected
patients can substantially improve upon treatment with Raxone. This
recommendation is a landmark in mitochondrial disease research worldwide and
will undoubtedly spur further research in this direction."
"We are very excited about the CHMP's positive opinion, which recognizes the
urgent medical need for a treatment for this devastating disease," stated Thomas
Meier, PhD, CEO of Santhera. "We can now execute on our plans to ensure Raxone
is made available to patients in the EU as soon as the European Commission
marketing authorization is received."
The CHMP based its recommendation for Raxone on data from the randomized,
placebo controlled RHODOS trial, Santhera's Expanded Access Program, and
comparative natural history data from a comprehensive case record survey. The
Committee considered that the totality of the data provided for an orphan
disease as severe as LHON with no available treatment options warranted a
recommendation for approval under Exceptional Circumstances. Santhera has
undertaken to gather additional long-term efficacy and safety data in LHON
patients as post-authorization measures.
The positive opinion by the CHMP for Raxone will now be forwarded to the
European Commission (EC) for the adoption of a decision on EU-wide marketing
authorization, applicable to all 28 member states of the European Union as well
as Iceland, Liechtenstein and Norway. Raxone has orphan designation which
provides 10 years of market exclusivity from the date of EC approval.
About Leber's Hereditary Optic Neuropathy and the therapeutic use of Raxone
Leber's Hereditary Optic Neuropathy (LHON) is a heritable genetic disease
causing blindness. The disease typically presents in young, otherwise healthy
adults, mostly men, as rapid, painless loss of central vision in one eye,
followed by visual loss in the other eye within a few months of the onset of
symptoms, leading to blindness. 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.
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 circumventing the complex I defect and restoring cellular energy
levels 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.
Santhera develops Raxone(®)/Catena(®) as treatment for patients with Leber's
Hereditary Optic Neuropathy (LHON), Duchenne Muscular Dys-trophy (DMD) and
primary progressive Multiple Sclerosis (ppMS) and omigapil for Congenital
Muscular Dystrophies (CMD), all areas of high unmet medical need. For further
information, please visit the Company's website www.santhera.com.
Raxone(®) and Catena(®) are trademarks of Santhera Pharmaceuticals.
For further information, contact:
Thomas Meier, PhD, Chief Executive Officer
Phone +41 61 906 89 64
thomas.meier(at)santhera.com
US investor contact:
Andrew McDonald, LifeSci Advisors, LLC
Phone +1 646 597 6979
andrew(at)lifesciadvisors.com
US Public Relations contact:
Deanne Eagle, Planet Communications
Phone +1 917 837 5866
deanne(at)planetcommunications.nyc
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 positive opinion:
http://hugin.info/137261/R/1931735/694768.pdf
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(ii) they are solely responsible for the content, accuracy and
originality of the information contained therein.
Source: Santhera Pharmaceuticals Holding AG via GlobeNewswire
[HUG#1931735]
Unternehmensinformation / Kurzprofil:
Datum: 26.06.2015 - 07:00 Uhr
Sprache: Deutsch
News-ID 403210
Anzahl Zeichen: 7593
contact information:
Town:
Liestal
Kategorie:
Business News
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