Santhera's Idebenone (Raxone) Receives Orphan Drug Designation for Duchenne Muscular Dystrophy in Australia
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Santhera's Idebenone (Raxone) Receives Orphan Drug Designation for Duchenne
Muscular Dystrophy in Australia
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The issuer is solely responsible for the content of this announcement.
Liestal, Switzerland, September 8, 2016 - Santhera Pharmaceuticals (SWX: SANN),
announces today that the Australian Therapeutic Goods Administration (TGA) has
granted orphan drug designation (ODD) to idebenone (Raxone) for the treatment of
Duchenne muscular dystrophy (DMD). The product has already received ODD from
European, Swiss and US authorities.
"We are delighted that the Australian authority has granted the orphan drug
designation for Raxone in DMD," commented Thomas Meier, PhD, CEO of Santhera.
"This decision underlines the need for a therapy for DMD and the potential role
Raxone might have as an effective treatment. We are grateful to the DMD patient
organization 'Save Our Sons-Duchenne Australia' which actively supported us in
this successful application."
Orphan drug legislation is designed to encourage pharmaceutical companies to
develop treatments for rare conditions. The TGA defines orphan diseases as
affecting fewer than 2000 individuals in Australia. Orphan drug designation is
an important first step in gaining regulatory approval and a drug with this
designation has market exclusivity in Australia of up to five years as per the
date of approval. The application for orphan drug designation was submitted on
Santhera's behalf by TudorRose Consulting Ltd, which is acting as Santhera's
legal sponsor in Australia.
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.
Following an exploratory Phase II trial (DELPHI), the safety and efficacy of
Raxone (idebenone) was investigated in the confirmatory phase III, double-blind,
placebo-controlled DELOS trial. DELOS 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. Results of the Phase II DELPHI trial were published by Buyse et
al. Neuromuscular Disorders 2011, 21: 396-405 and Pediatric Pulmonology
2013, 48: 912-20. The positive outcome of the Phase III DELOS study was
published by Buyse et al., The Lancet 2015, 385: 1748-1757 and McDonald et al.,
Neuromuscular Disorders 2016, 26: 473-480.
The European Medicines Agency's Committee for Medicinal Products for Human Use
(CHMP) is currently assessing a Marketing Authorization Application (MAA) for
Raxone in DMD patients with respiratory function decline who are not taking
concomitant glucocorticoids. The indication would include patients who
previously were treated with glucocorticoids or in whom glucocorticoid treatment
is not desired, not tolerated or is contraindicated. The MAA was submitted as a
Type II variation of the company's existing marketing authorization for Raxone
for the treatment of visual impairment in patients with Leber's hereditary optic
neuropathy (LHON).
About TudorRose Consulting Pty Ltd
TudorRose Consulting Pty Ltd has been operating as a Regulatory and Quality
consulting company, based in Melbourne, Australia since July 2010. The company
is specialized in international product development and provides a broad range
of services including Regulatory Affairs, QA, Project Management and Strategy.
For further information, please visit the Company's website
www.tudorroseconsulting.com.au.
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 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
Daniel Piller, Head Communications
Phone +41 61 906 89 26
daniel.piller(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 press release 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 Orphan Designation Australia:
http://hugin.info/137261/R/2040659/761263.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: 08.09.2016 - 07:00 Uhr
Sprache: Deutsch
News-ID 493430
Anzahl Zeichen: 8164
contact information:
Town:
Liestal
Kategorie:
Business News
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