Santhera's Marketing Authorization Application for Raxone® in Duchenne Muscular Dystrophy (DMD) Validated by the European Medicines Agency
(Thomson Reuters ONE) -
Santhera Pharmaceuticals Holding AG /
Santhera's Marketing Authorization Application for Raxone® in Duchenne Muscular
Dystrophy (DMD) Validated by the European Medicines Agency
. Processed and transmitted by NASDAQ OMX Corporate Solutions.
The issuer is solely responsible for the content of this announcement.
Liestal, Switzerland, June 21, 2016 - Santhera Pharmaceuticals (SIX: SANN)
announces that the European Medicines Agency (EMA) has validated its Marketing
Authorization Application (MAA) for Raxone(®) for Duchenne muscular dystrophy
(DMD) in patients with respiratory function decline who are not taking
concomitant glucocorticoids. Validation confirms that the submission, which was
filed as Type II variation of the Company's existing marketing authorization for
Raxone, is complete and that the review process by the CHMP (Committee for
Medicinal Products for Human Use) has begun. Santhera expects an opinion from
the CHMP in the first quarter of 2017.
The regulatory dossier was submitted as a Type II Variation of the existing
marketing authorization and is based on data from Santhera's phase II (DELPHI)
program and the successful pivotal phase III (DELOS) study which enrolled
patients not taking concomitant glucocorticoids. These data, which demonstrated
a statistically significant and clinically relevant benefit of Raxone treatment
in preserving respiratory function compared to placebo, have been substantiated
by a natural history study showing that the benefit observed in the Raxone-
treated group would not have been expected from the natural course of the
disease. The MAA also includes recently published data that demonstrated that
patients receiving Raxone in the DELOS trial had a reduced risk of
bronchopulmonary complications including fewer hospitalizations caused by such
complications and a reduced need for systemic antibiotic treatment compared to
patients receiving placebo. The clinical relevance of these findings is
supported by the outcome of a patient-centered benefit-risk survey which showed
that DMD patients and their caregivers placed a high value on treatments that
could reduce pulmonary complications.
"Validation of our submission by the EMA acknowledges the completeness of our
dossier which combines data from our clinical program and supporting natural
history data together establishing the clinical meaningfulness of Raxone
treatment in DMD," commented Thomas Meier, PhD and CEO of Santhera.
"Preservation of respiratory function is a major goal of DMD therapy and the
initiation of the CHMP review of our MAA is an important milestone for patients
with DMD who currently have no alternative treatment."
Raxone was approved in September 2015 for the treatment of visual impairment in
adolescent and adult patients with Leber's hereditary optic neuropathy (LHON) in
all EU member states, Norway, Iceland and Liechtenstein.
About Raxone(®) (Idebenone), Duchenne Muscular Dystrophy and DELOS
Duchenne muscular dystrophy (DMD) is one of the most common and devastating
types of muscle degeneration and results in rapidly progressive muscle weakness.
DMD is characterized by a loss of the protein dystrophin, leading to cell
damage, impaired calcium homeostasis, elevated oxidative stress and reduced
energy production in muscle cells. This results in progressive muscle weakness
and wasting and early morbidity and mortality due to respiratory failure.
Idebenone is a synthetic short-chain benzoquinone and a cofactor for the enzyme
NAD(P)H:quinone oxidoreductase (NQO1) capable of stimulating mitochondrial
electron transport, reducing and scavenging reactive oxygen species (ROS) and
supplementing cellular energy levels.
DELOS was a phase III, double-blind, placebo-controlled trial which randomized
64 patients, not taking concomitant glucocorticoids, to receive either Raxone
tablets or matching placebo. The trial met its primary endpoint and demonstrated
that Raxone can slow the loss of respiratory function and reduces
bronchopulmonary complications. The positive outcome of the phase III DELOS
study was first published in The Lancet (Buyse et al., The Lancet
2015 385(9979):1748-57) and in Neuromuscular Disorders (article in press
http://dx.doi.org/10.1016/j.nmd.2016.05.008).
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's lead product Raxone(®) is authorized in the European Union for the
treatment of Leber's hereditary optic neuropathy (LHON). For Duchenne muscular
dystrophy (DMD), the second indication for Raxone(®), Santhera has filed a
Marketing Authorization Application (MAA) in the European Union. Santhera
develops Raxone(®) in a third indication, primary progressive multiple sclerosis
(PPMS), and omigapil for congenital muscular dystrophy (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 Christoph Rentsch, Chief
Financial Officer
Phone +41 61 906 89 64 Phone
+41 61 906 89 65
thomas.meier(at)santhera.com
christoph.rentsch(at)santhera.com
US investor contact US Public Relations
contact
Hans Vitzthum, LifeSci Advisors, LLC Deanne Eagle, Planet
Communications
Phone +1 212 915 2568 Phone
+1 917 837 5866
hans(at)lifesciadvisors.com
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 EMA Validation:
http://hugin.info/137261/R/2021954/751226.pdf
This announcement is distributed by GlobeNewswire on behalf of
GlobeNewswire clients. The owner of this announcement warrants that:
(i) the releases contained herein are protected by copyright and
other applicable laws; and
(ii) they are solely responsible for the content, accuracy and
originality of the information contained therein.
Source: Santhera Pharmaceuticals Holding AG via GlobeNewswire
[HUG#2021954]
Unternehmensinformation / Kurzprofil:
Datum: 21.06.2016 - 07:00 Uhr
Sprache: Deutsch
News-ID 478917
Anzahl Zeichen: 8109
contact information:
Town:
Liestal
Kategorie:
Business News
Diese Pressemitteilung wurde bisher 323 mal aufgerufen.
Die Pressemitteilung mit dem Titel:
"Santhera's Marketing Authorization Application for Raxone® in Duchenne Muscular Dystrophy (DMD) Validated by the European Medicines Agency"
steht unter der journalistisch-redaktionellen Verantwortung von
Santhera Pharmaceuticals Holding AG (Nachricht senden)
Beachten Sie bitte die weiteren Informationen zum Haftungsauschluß (gemäß TMG - TeleMedianGesetz) und dem Datenschutz (gemäß der DSGVO).
