Swissmedic Accepts Santhera's Filing of SNT-MC17 in Friedreich's
Ataxia
Liestal and Lachen, Switzerland, October 4, 2007 - Santhera
Pharmaceuticals (SWX:SANN, "Santhera"), a Swiss specialty
pharmaceutical company focused on neuromuscular diseases, and Takeda
Pharma AG ("Takeda"), jointly announced today that Swissmedic, the
Swiss regulatory authority, has accepted the filing of an application
for authorization for Santhera's lead compound SNT-MC17 (INN:
idebenone, originally developed by Takeda) for the treatment of
Friedreich's Ataxia (FRDA). SNT-MC17 has shown clinical efficacy in
FRDA patients on neurological and cardiac endpoints in several
clinical trials and proved to be well tolerated in all studies so
far. Upon approval, the product will be marketed in Switzerland by
Santhera's partner Takeda. The acceptance of the marketing
authorization application in Switzerland follows shortly after the
application for marketing authorization in the EU.
The dossier submitted to Swissmedic includes efficacy data generated
in the collaborative study with the US National Institutes of Health
(NIH) analyzing a variety of neurological and cardiac outcome
measures. The file is supported by data from earlier clinical trials
in FRDA conducted by academic institutions that demonstrated efficacy
primarily in the treatment of the cardiac symptoms of this
devastating disease. The safety package consists of data generated by
Santhera with SNT-MC17 and Takeda in its earlier preclinical and
clinical development program with idebenone. The Swiss application
for authorization recommends a starting dose of 450 mg/day for
patients below 45 kg body weight and 900 mg/day for patients of 45 kg
or above body weight, with the option for the treating physician to
use higher doses if needed. The dosing recommendations as well as the
data package of the Swiss filing are equivalent to the EMEA filing.
There is an estimated population of 100 to 200 FRDA patients living
in Switzerland. An application for orphan drug status has been filed
with Swissmedic but will be reviewed independently. Santhera and
Takeda believe that the compound has the potential to be granted
marketing approval in Switzerland for the treatment of FRDA in the
second half of 2008.
Upon marketing approval, SNT-MC17 will be distributed by Takeda
Pharma AG. In Switzerland, Takeda has obtained a temporary
authorization in May 2004 for its idebenone, which is fully
reimbursed to treat cardiomyopathy in FRDA patients. This temporary
status will be revoked once SNT-MC17 is approved and Takeda will
subsequently switch to Santhera's product.
Klaus Schollmeier, Santhera's CEO, commented: "We are excited about
Swissmedic's acceptance of our marketing authorization application.
SNT-MC17 has shown clinically relevant improvements of neurological
and cardiac symptoms associated with FRDA. If everything goes
according to plan, we may be able to launch SNT-MC17 in Switzerland
still in 2008, thus ensuring a timely transition from the temporary
to the full registration".
Jean-Luc Delay, CEO of Takeda Pharma AG, said: "We have been able to
bring notable benefit to Swiss FRDA patients on the basis of
idebenone's temporary authorization. Thanks to our joint efforts with
Santhera, we now look forward to extend the patient population
eligible for this first, fully approved therapeutic therapy. In
addition, FRDA patients could benefit from the higher dosing option
of SNT-MC17 as supported by clinical evidence."
About Friedreich's Ataxia (FRDA)
Friedreich's Ataxia (FRDA) is a rare but severe genetic neuromuscular
disorder that results in the degeneration of an individual's nerve
and muscle tissue. This disorder causes loss of muscle control,
uncoordinated movements, muscle wasting and thickening of heart walls
which frequently leads to a shortened life span. FRDA affects both
Caucasian males and females equally and it is estimated that about
20,000 patients suffer from the disease in both North America and
Europe. Average life expectancy for FRDA patients is limited to
approximately 35 to 50 years.
The disorder results from a genetic defect in the gene encoding for
frataxin. Reduced levels of this protein ultimately result in
impaired energy production in mitochondria, the cells' energy
production centers, and elevated oxidative stress. Tissues that have
the highest need for energy, in particular nerve and cardiac tissues,
are primarily affected by frataxin deficiency resulting in
pathological changes in heart muscle anatomy and function and loss of
nerve cells. SNT-MC17 is believed to improve the balance and flow of
electrons within the mitochondria, therefore increasing the energy
production within nerve and muscle cells of FRDA patients, protecting
these cells from cell death. A number of clinical trials have
provided strong evidence that SNT-MC17 may offer an effective
treatment option for FRDA associated heart wall thickening
(cardiomyopathy). In addition, data from the collaborative NIH
clinical trial suggest positive effects on neurological function.
* * *
About Santhera
Santhera Pharmaceuticals (SWX: SANN) is a Swiss specialty
pharmaceutical company focused on the discovery, development and
marketing of small-molecule pharmaceutical products for the treatment
of severe neuromuscular diseases. Santhera's vision is to become a
leading specialty pharmaceutical company offering therapies for a
number of indications in this area of high unmet medical need which
includes many orphan indications with no current therapy.
Santhera currently has five clinical-stage development programs,
three of which are investigating its lead compound, SNT-MC17 (INN:
idebenone), for the treatment of Friedreich's Ataxia (FRDA), Duchenne
Muscular Dystrophy (DMD) and Leber's Hereditary Optic Neuropathy
(LHON). Another clinical program is investigating JP-1730 (INN:
fipamezole) for the treatment of Dyskinesia in Parkinson's Disease
(DPD) in cooperation with Juvantia, the compound's owner. The fifth
program comprises SNT-317 (INN: omigapil) in Congenital Muscular
Dystrophies (CMD), a compound in-licensed from Novartis. The most
advanced program, SNT-MC17 in FRDA, is currently under review for
marketing approval in the EU and will be submitted shortly in Canada.
The compound is also in Phase III clinical development for FRDA in
the US while the while the other clinical programs are in Phase II.
For further information, please visit www.santhera.com.
About Takeda Pharma AG
Takeda Pharma AG is one of the top 20 pharmaceutical companies in
Switzerland, and one of the fastest growing. It is a subsidiary of
Takeda Pharmaceuticals Corporation, Japan (TSE:4502), a
research-based global company with its main focus on pharmaceuticals.
As the largest pharmaceutical company in Japan and one of the global
leaders of the industry, Takeda is committed to striving toward
better health for individuals and progress in medicine by developing
superior pharmaceutical products.
Aiming to become an "R&D-driven world-class pharmaceutical company",
Takeda is enhancing its R&D pipeline by concentrating its management
resources for that purpose in the following selected core therapeutic
areas:
* cardio-metabolic diseases,
* oncology and urological diseases
* central nervous system disorders, bone/joint diseases
* gastroenterological diseases
Additional information about Takeda Pharma is available through its
website, www.takeda.ch.
For further information, contact
Santhera Pharmaceuticals
Klaus Schollmeier, Chief Executive Officer
Phone: +41 (0)61 906 89 52
klaus.schollmeier@santhera.com
Barbara Heller, Chief Financial Officer
Phone: +41 (0)61 906 89 54
barbara.heller@santhera.com
Thomas Staffelbach, VP Public & Investor Relations
Phone: +41 (0)61 906 89 47
thomas.staffelbach@santhera.com
Takeda Pharma AG
Jean-Luc Delay, Chief Executive Officer
Phone: +41 (0)55 451 52 21
jean-luc.delay@takeda.ch
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--- End of Message ---
Santhera Pharmaceuticals Holding AG
Hammerstrasse 47 Liestal
Switzerland
WKN: A0LCUK; ISIN: CH0027148649; Index: SPI, SPIEX, SSCI;
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