Santhera Files Marketing Authorization Application in the European Union for Raxone® for the Treatment of Duchenne Muscular Dystrophy
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Santhera Pharmaceuticals Holding AG /
Santhera Files Marketing Authorization Application in the European Union for
Raxone® for the Treatment of Duchenne Muscular Dystrophy
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Liestal, Switzerland, May 31, 2016 - Santhera Pharmaceuticals (SIX: SANN)
announces that it has submitted a Marketing Authorization Application (MAA) to
the European Medicines Agency (EMA) for Raxone(®) (idebenone) for the treatment
of Duchenne muscular dystrophy (DMD) in patients with respiratory function
decline and not taking concomitant glucocorticoids. The new indication was
submitted as Type II variation of the company's existing marketing authorization
for Raxone granted last year. Raxone has Orphan Drug Designation for DMD in the
EU.
The regulatory dossier now submitted as Type II Variation of the existing
marketing authorization summarizes data from Santhera's phase II (DELPHI)
program and the successful pivotal phase III (DELOS) study which demonstrated a
statistically significant and clinically relevant benefit of Raxone treatment in
slowing the rate of respiratory function loss compared to placebo in patients
with DMD not taking concomitant glucocorticoids. This benefit also translated to
fewer bronchopulmonary events (e.g. airway infections), shorter event duration
and less antibiotic treatment in the Raxone-treated patients. The data have been
substantiated by a natural history study showing that the significant decline of
respiratory function observed in the placebo group of DELOS reflected the
natural course of the disease whilst the outcome for Raxone-treated patients did
not. The MAA also includes data from a patient-centered benefit-risk survey
which highlighted the importance of treating pulmonary disease in patients with
DMD. The findings showing that Raxone slows the rate of respiratory function
decline, decreases the proportion of patients crossing clinically relevant
functional thresholds and increases the time until such thresholds are reached,
represent an important treatment effect and are of major clinical relevance for
patients with DMD. Raxone (900 mg/day) was safe and well tolerated with adverse
event rates comparable to placebo.
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. The indication would include patients who previously were
treated with glucocorticoids or in whom glucocorticoid treatment is not desired,
not tolerated or is contraindicated. Patients with respiratory function decline
currently not using glucocorticoids account for approximately 40% of DMD
patients above the age of 10 years. There is currently no treatment available
for this group of DMD patients.
"Raxone provides clinically relevant benefit across multiple pulmonary function
parameters as shown in our DELOS trial, the first and to date only double-blind,
placebo-controlled phase III trial in DMD with a positive outcome", said Thomas
Meier, PhD, CEO of Santhera. "We are now working diligently with European
regulators to provide rapid access to Raxone for patients with respiratory
function loss, who currently have no effective treatment options".
In the USA, Santhera has submitted comprehensive briefing material to the FDA in
preparation for discussion on the filing of a New Drug Application (NDA) for
idebenone under Subpart H for the same indication as in the MAA. Feedback from
the FDA is currently expected by the end of July.
Santhera holds global commercialization rights to the DMD program which has been
granted Orphan Drug Designation in the EU and the US and Fast Track Designation
in the US. Patent protection extends until March 2026 (EU, Japan) and December
2027 (USA).
About Raxone(®) (Idebenone) as Treatment of Duchenne Muscular Dystrophy (DMD)
Duchenne muscular dystrophy (DMD) is one of the most common and devastating
types of muscle degeneration and results in rapidly progressive muscle weakness.
It is a genetic, degenerative disease that is inherited in an X-linked recessive
mode with an incidence of up to 1 in 3,500 live born males worldwide. 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, 10-18 years of age, 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 in patients not taking concomitant
glucocorticoids. The positive outcome of the phase III DELOS study was published
in The Lancet (Buyse et al., The Lancet 2015 385(9979):1748-57).
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). Santhera develops
Raxone(®) in two additional indications, Duchenne muscular dystrophy (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 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 MAA submission EU:
http://hugin.info/137261/R/2016612/747990.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#2016612]
Unternehmensinformation / Kurzprofil:
Datum: 31.05.2016 - 07:00 Uhr
Sprache: Deutsch
News-ID 474418
Anzahl Zeichen: 9109
contact information:
Town:
Liestal
Kategorie:
Business News
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