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Santhera Pharmaceuticals Holding AG: Santhera receives FDA Fast Track Designation for Raxone®/Catena® (idebenone) for theTreatment of Duchenne Muscular Dystr

Santhera Pharmaceuticals Holding AG / Santhera receives FDA Fast Track
Designation for Raxone®/Catena® (idebenone) fortheTreatment of Duchenne
Muscular Dystrophy . Processed and transmitted by NASDAQ OMX Corporate
Solutions.The issuer is solely responsible for the content of this
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

"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," commentedThomas 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

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

Raxone®and Catena®are trademarks of Santhera Pharmaceuticals.

# # #

| For further information, contact: |
| Thomas Meier, Chief Executive Officer |
| Phone: +41 61 906 89 64 |
| |
| |
| 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 |
| |
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


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


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