Santhera Updates on Regulatory Filings for Raxone® (idebenone) in Duchenne Muscular Dystrophy (DMD)
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Santhera Pharmaceuticals Holding AG /
Santhera Updates on Regulatory Filings for Raxone® (idebenone) in Duchenne
Muscular Dystrophy (DMD)
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Liestal, Switzerland, May 3, 2016 - Santhera Pharmaceuticals (SIX: SANN)
announces that it has submitted comprehensive briefing material and a meeting
request to the FDA to discuss the filing of a New Drug Application (NDA) for
Raxone(®) (idebenone) for the treatment of DMD patients not taking concomitant
glucocorticoids. A Marketing Authorization Application for DMD in Europe is also
expected to be submitted in coming weeks. The Company also reports that, as part
of its strategy to expand the Raxone label, a new phase III study (SIDEROS) in
DMD patients using glucocorticoids will start enrolling patients in coming
weeks.
The FDA-meeting request included a very comprehensive data package intended to
prepare for discussions with the Agency on an accelerated NDA approval (under
Subpart H) for Raxone in patients with DMD not taking concomitant
glucocorticoids. The intended indication is for patients in whom respiratory
function has started to decline and would include patients who previously were
treated with glucocorticoids or in whom glucocorticoid treatment is not desired,
not tolerated or is contraindicated.
The data package provided to the FDA summarizes data from Santhera's phase II
(DELPHI) program and the successful pivotal phase III (DELOS) study, which
demonstrated a clinically relevant and statistically significant benefit of
idebenone treatment in slowing the rate of respiratory function decline compared
to placebo. Importantly, the package also includes data from the Cooperative
International Neuromuscular Research Group's (CINRG) Duchenne natural history
study (DNHS), which, in collaboration with CINRG, were used to conduct the first
prospectively planned external control group study to compare outcomes for
patients participating in DELOS with matched, contemporaneously-observed
patients from the CINRG DNHS. The results demonstrate that the respiratory
function decline observed in the placebo group of the DELOS study is consistent
with the rate of decline observed in matched patients from the CINRG DNHS and
therefore with the expected natural history of DMD. However, the slower rate of
decline observed in idebenone-treated patients in DELOS was not observed in
matched patients from the CINRG DNHS, indicating that the rate of respiratory
function decline in idebenone-treated patients in DELOS differs from the
expected natural history of DMD.
"We foresaw the FDA requirement for prospective planning of the natural history
control group matching process and have been working with CINRG since last year
to provide such a study in support of our NDA dossier" commented Thomas Meier,
PhD, CEO of Santhera. "The successful outcome of this prospectively-planned,
matched-patient study is unprecedented in clinical research in DMD and provides
additional external validation of the results of our successful DELOS study".
Submission documents using the same data sets are currently being finalized for
a Type II-variation of the existing Marketing Authorization granted for Raxone
for Leber's hereditary optic neuropathy in Europe to add the treatment of DMD
patients not using glucocorticoids to the label.
The Company also reported that the SIDEROS protocol for a new phase III study in
DMD patients using glucocorticoids has successfully completed the FDA review
process. The Company will make a further announcement as soon as the study
starts enrolling patients. Patients with declining respiratory function on any
stable glucocorticoid treatment scheme will be eligible. Study participants will
receive either Raxone (900 mg/day) or placebo for 78 weeks. The trial targets to
enroll approximately 260 DMD patients and will be conducted in Europe and the
US. Patients completing the trial will be offered the opportunity to enroll in
an open label extension study. Santhera intends to position this study to
provide confirmatory evidence (under Subpart H) of the efficacy of idebenone in
patients both taking and not taking concomitant glucocorticoids.
"Following the successful outcome of our DELOS study, a further placebo-
controlled study in patients not taking glucocorticoids is not considered
feasible. However, patients taking glucocorticoids represent a different but
related population that is capable of verifying predicted clinical benefit and
in which a successful outcome can be considered confirmation of clinical benefit
in DMD patients with respiratory function decline, irrespective of
glucocorticoid use", commented Nicholas Coppard, PhD, Head of Development at
Santhera.
About 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, 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 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 DMD filing update:
http://hugin.info/137261/R/2009150/743460.pdf
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originality of the information contained therein.
Source: Santhera Pharmaceuticals Holding AG via GlobeNewswire
[HUG#2009150]
Unternehmensinformation / Kurzprofil:
Datum: 03.05.2016 - 07:00 Uhr
Sprache: Deutsch
News-ID 468067
Anzahl Zeichen: 10017
contact information:
Town:
Liestal
Kategorie:
Business News
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