Swissmedic Accepts for Review Santhera's Marketing Authorization Application for Raxone® for the Treatment of Duchenne Muscular Dystrophy
(Thomson Reuters ONE) -
Santhera Pharmaceuticals Holding AG /
Swissmedic Accepts for Review Santhera's Marketing Authorization Application for
Raxone® for the Treatment of Duchenne Muscular Dystrophy
. Processed and transmitted by Nasdaq Corporate Solutions.
The issuer is solely responsible for the content of this announcement.
Liestal, Switzerland, November 8, 2016 - Santhera Pharmaceuticals (SIX: SANN)
announces that the Swiss Agency for Therapeutic Products (Swissmedic) has
accepted for review Santhera's Marketing Authorization Application (MAA) for
Raxone(®) (idebenone) for the treatment of Duchenne muscular dystrophy (DMD) in
patients with respiratory function decline not taking concomitant
glucocorticoids. Raxone was granted Orphan Drug Designation for DMD in
Switzerland in 2012.
Santhera submitted a MAA to Swissmedic for Raxone in DMD in October 2016.
Swissmedic has now confirmed that the dossier is sufficiently complete to permit
substantive review. The standard assessment time is typically 15-18 months. The
MAA for Raxone is already under review for the same indication by the Committee
for Medicinal Products for Human Use (CHMP) of the European Medicines Agency
(EMA).
"This submission reflects our continued efforts to make Raxone available to
patients and physicians beyond the European Union," said Thomas Meier, PhD, CEO
of Santhera. "We look forward to Swissmedic's review of our application and to
the approval of Raxone in Switzerland for DMD patients with respiratory function
loss."
The intended indication for Raxone is for patients with DMD in whom respiratory
function has started to decline and who are currently not taking concomitant
glucocorticoids. This indication would include patients who were previously
treated with glucocorticoids or in whom glucocorticoid treatment is not desired,
not tolerated or is contraindicated.
As part of the Swiss MAA, Santhera submitted data from its phase II (DELPHI)
program and the successful pivotal phase III (DELOS) study, the results of which
have been further substantiated by a comparative natural history study. Data
from all studies demonstrate that Raxone slows the rate of respiratory function
decline compared to untreated patients to a degree that is of major clinical
relevance for patients with DMD. Raxone (900 mg/day) was safe and well tolerated
with adverse event rates comparable to placebo.
In parallel to the already ongoing MAA reviews in Europe, Santhera continues to
prepare for further discussions with the United States' Food and Drug
Administration on the most appropriate regulatory pathway to approval in the US.
About Raxone(®) (Idebenone) in Duchenne Muscular Dystrophy
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
(900 mg/day) 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 statistically significant and clinically
relevant outcomes of the phase III DELOS study were published: Buyse et al., The
Lancet 2015, 385:1748-1757; McDonald et al. Neuromuscular Disorders
2016, 26: 473-480 and Buyse et al., Pediatric Pulmonology 2016:
http://dx.doi.org/10.1002/ppul.23547.
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, Norway,
Iceland and Liechtenstein 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. In collaboration with the US National Institute of
Neurological Disorders and Stroke (NINDS) Santhera is developing 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(®) is a trademark of Santhera Pharmaceuticals.
For further information, contact:
Thomas Meier, PhD, Chief Executive Christoph Rentsch, Chief Financial
Officer 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 MAA CH:
http://hugin.info/137261/R/2054999/769437.pdf
This announcement is distributed by Nasdaq Corporate Solutions on behalf of Nasdaq 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
Unternehmensinformation / Kurzprofil:
Datum: 08.11.2016 - 07:00 Uhr
Sprache: Deutsch
News-ID 505481
Anzahl Zeichen: 7782
contact information:
Town:
Liestal
Kategorie:
Business News
Diese Pressemitteilung wurde bisher 282 mal aufgerufen.
Die Pressemitteilung mit dem Titel:
"Swissmedic Accepts for Review Santhera's Marketing Authorization Application for Raxone® for the Treatment of Duchenne Muscular Dystrophy"
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).
