Bli medlem
Bli medlem

Du är här

2016-05-20

Santhera Pharmaceuticals Holding AG: Santhera receives FDA Fast Track Designation for Omigapil for the Treatment of Congenital Muscular Dystrophies (CMD)

Santhera Pharmaceuticals Holding AG / Santhera receives FDA Fast Track
Designation for Omigapil for the Treatment ofCongenital Muscular Dystrophies
(CMD) . Processed and transmitted by NASDAQ OMX Corporate Solutions.The
issuer is solely responsible for the content of this announcement.
Liestal, Switzerland, May 20, 2015 - Santhera Pharmaceuticals (SIX: SANN)
announces that it has received Fast Track Designation from the US Food and
Drug Administration (FDA) for omigapil for the treatment of congenital
muscular dystrophies (CMD).
FDA's Fast Track process is designed to facilitate the development and
expedite the review of drugs to treat serious conditions and fill an unmet
medical need for the purpose of getting them to the patient earlier.
Previously, omigapil has already been granted Orphan Drug Designation for CMD
in both the EU and the US. Santhera, in collaboration with the US National
Institutes of Health (NIH), is currently conducting a clinical phase I study
(CALLISTO) with omigapil in CMD patients.

"We are delighted that the FDA has granted Fast Track Designation for omigapil
thereby underlining the unmet medical need for an effective therapy for CMD
patients and the severity of this class of diseases," commentedThomas Meier
, PhD, CEO of Santhera. "After Raxone®, omigapil is our second neuromuscular
pipeline product and underpins our dedication to developing effective
medicines to satisfy the needs of patients suffering from mitochondrial and
other rare diseases."

CMD is a group of inherited, severe neuromuscular diseases characterized by
different forms of progressive and ultimately devastating loss of muscle
tissue, frequently affecting young children. Currently, no treatment is
available to slow down or stop progression of the disease. Santhera evaluates
omigapil in the phase I study (CALLISTO) which investigates the safety,
tolerability and pharmacokinetic profile of a new liquid formulation of
omigapil in pediatric and adolescent patients with CMD. The study, which is
being conducted at the US NIH's National Institute of Neurological Disorders
and Stroke (NINDS) in Bethesda, Maryland, with Prof.Carsten Bönnemann
as Principal Investigator, is fully enrolled and expected to be completed
early in 2017. As previously reported, the CALLISTO study is supported
financially by a public-private partnership including two patient
organizations, the US-based Cure CMD and the Swiss Foundation for Research on
Muscle Diseases, and EndoStem, an EU 7th Framework program.

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 Congenital Muscular Dystrophies

Congenital muscular dystrophies (CMD) refer to a variety of inherited
neuromuscular conditions characterized by different forms of progressive loss
of muscle tissue. Severe forms can affect newborns or young children with
life-threatening progressive muscle weakness ("floppy infant syndrome").
Complications associated with the disorder such as loss of body weight,
skeletal deformations and respiratory distress result in immobility at young
age and early mortality. No pharmacological therapy is currently available or
in advanced clinical development. Treatment options are confined to
respiratory support and orthopedic surgery for scoliosis as well as
supplementary nutrition to avoid malnutrition.

About Omigapil

Omigapil is a deprenyl-analog with anti-apoptotic properties, originally
developed by Novartis. Santhera obtained an exclusive license for omigapil
for the development in congenital muscular dystrophies (CMD). Nonclinical
studies in a disease-relevant model showed that omigapil inhibits cell death
and reduces body weight loss and skeletal deformation, while increasing
locomotive activity and protecting from early mortality. Clinical development
of omigapil is sponsored by Santhera under an open IND granted by the US Food
and Drug Administration.

About CALLISTO

CALLISTO is a phase I study evaluating the pharmacokinetics, safety and
tolerability of omigapil in 20 ambulatory and non-ambulatory patients aged 5
to 16 years affected by either Ullrich or MDC1A subtypes of CMD. The study is
being conducted at the NIH's National Institute of Neurological Disorders and
Stroke (NINDS) in Bethesda, Maryland, and has been fully enrolled. More
details on the study are available fromwww.clinicaltrials.gov(Identifier
NCT01805024).

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
websitewww.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@santhera.com
christoph.rentsch@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@lifesciadvisors.com
deanne@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 Fast Track Status Omigapil
http://hugin.info/137261/R/2014072/746585.pdf

---------------------------------------

This announcement is distributed by NASDAQ OMX Corporate Solutions on behalf of NASDAQ OMX 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

HUG#2014072

Författare Hugin

Tala om vad ni tycker

Tala om vad ni tycker

Ni är just nu inne på en betaversion av nya aktiespararna. Lämna gärna feedback på vad ni tycker i formuläret nedan.