Santhera receives FDA Fast Track Designation for Raxone®/Catena® (idebenone) for theTreatment of Duchenne Muscular Dystrophy
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Santhera Pharmaceuticals Holding AG /
Santhera receives FDA Fast Track Designation for Raxone®/Catena® (idebenone) for
theTreatment of Duchenne Muscular Dystrophy
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The issuer is solely responsible for the content of this announcement.
Liestal, Switzerland, April 9, 2015 - Santhera Pharmaceuticals (SIX: SANN)
announces that the U.S. Food and Drug Administration (FDA) has granted Fast
Track designation for Santhera's Raxone(®)/Catena(®) (idebenone) for the
treatment of Duchenne Muscular Dystrophy (DMD). FDA's Fast Track program
facilitates the development and review of important drugs intended to treat
serious conditions and fill an unmet medical need for the purpose of getting
them to the patient earlier. Santhera previously announced that the Phase III
trial (DELOS) in DMD met its primary endpoint and demonstrated that
Raxone/Catena delayed the loss of respiratory function.
"We are very pleased that the FDA has granted Fast Track designation for
Raxone/Catena, which further underscores the unmet medical need for effective
treatments for patients with DMD," commented Thomas Meier, PhD, CEO of Santhera.
"On the basis of the positive data from our Phase III trial with Raxone/Catena
in DMD, we have started to prepare a New Drug Application and plan to meet with
the FDA in the coming weeks to discuss our NDA dossier in a pre-NDA meeting."
About FDA Fast Track Designation
The FDA established the Fast Track Drug Development Program under the FDA
Modernization Act of 1997. The program is designed to facilitate the development
and expedite the review of therapies intended to treat serious or life-
threatening conditions, and that demonstrate the potential to address unmet
medical needs. The advantages of Fast Track designation include more frequent
meetings with the FDA, eligibility for Accelerated Approval and Priority Review,
if supported by clinical data and Rolling Review, which allows a company to
submit its NDA in sections, as they are completed. Usually the FDA does not
begin review until it has received a complete application.
About Duchenne Muscular Dystrophy (DMD)
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 cardio-respiratory failure. Currently, glucocorticoid steroids
are the only available medical treatment that can slow the decline in muscle
strength and function, irrespective of the disease-causing mutation. However,
the effect is only partial and clinical use is limited by well-known side
effects caused by steroids. A recent study showed that ~42% of DMD patients 10
years and older had either never used steroids or have discontinued their use.
About Idebenone in Duchenne Muscular Dystrophy
Raxone/Catena (idebenone) is a synthetic short-chain benzoquinone and a
substrate for the enzyme NAD(P)H:quinone oxidoreductase (NQO1) capable of
stimulating mitochondrial electron transport and supplementing cellular energy
levels. A prior Phase II randomized placebo-controlled trial (DELPHI)
demonstrated trends for beneficial effects of Raxone/Catena on early functional
cardiac and respiratory parameters. An important finding of the DELPHI trial was
that patients treated with idebenone stabilized in PEF%p, a marker of expiratory
muscle strength, compared to patients receiving placebo who declined as expected
from the natural history of the disease. Additional ana-lyses indicated that the
Raxone/Catena treatment effect on respiratory function outcomes was larger in
patients not taking concomitant glucocorticoid steroids.
Idebenone has been granted orphan drug designation for DMD in Europe and the US
and has use patent protection until 2026 in Europe and 2027 in the US.
About the DELOS trial
DELOS was a Phase III, double-blind, placebo-controlled trial which randomized
and treated 64 European and US DMD patients not receiving concomitant
corticosteroids. Patients 10-18 years of age received either Raxone/Catena
tablets (900 mg/day) or matching placebo for 52 weeks. The primary endpoint was
change in Peak Expiratory Flow % predicted (PEF%p) from baseline to week 52.
PEF%p declined significantly (-9.01%p; 95% CI: -13.2, -4.8; p<0.001) from
baseline to week 52 in the placebo group compared to a non-significant decline
(-3.05%p; 95% CI: -7.1, 0.97; p=0.134) in the Raxone/Catena group, resulting in
a statistically significant difference between treatment groups of 5.96%p (95%
CI: 0.16, 11.8; p=0.044) at week 52 and representing a 66% reduction in loss of
PEF%p. A statistically significant treatment effect was also seen at week 26
(p=0.007) and week 39 (p=0.034) and across all assessment timepoints (p=0.018).
Data for the primary endpoint were robust across multiple sensitivity analyses
and supported by positive outcomes of additional respiratory endpoints.
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 develops Raxone(®)/Catena(®) as treatment for patients with Leber's
Hereditary Optic Neuropathy (LHON), 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 which no
therapies are currently available. 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, Chief Executive Officer
Phone: +41 61 906 89 64
thomas.meier(at)santhera.com
US investor contact: US Public Relations contact:
Andrew McDonald, LifeSci Advisors, LLC Deanne Eagle, Planet Communications
Phone: +1 646 597 6979 Phone: +1 917 837 5866
andrew(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 FDA fast track:
http://hugin.info/137261/R/1909640/680846.pdf
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originality of the information contained therein.
Source: Santhera Pharmaceuticals Holding AG via GlobeNewswire
[HUG#1909640]
Unternehmensinformation / Kurzprofil:
Datum: 09.04.2015 - 07:15 Uhr
Sprache: Deutsch
News-ID 384442
Anzahl Zeichen: 8716
contact information:
Town:
Liestal
Kategorie:
Business News
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