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Santhera Receives Rare Pediatric Disease Designation from FDA for Idebenone for the Treatment of Duchenne Muscular Dystrophy


August 19, 2015 - London

Santhera Pharmaceuticals Holding AG /Santhera Receives Rare Pediatric Disease Designation from FDA for Idebenone for the Treatment of Duchenne Muscular Dystrophy . Processed and transmitted by NASDAQ OMX Corporate Solutions.The issuer is solely responsible for the content of this announcement.

Liestal, Switzerland, August 19, 2015 - Santhera Pharmaceuticals (SIX: SANN) announces that the U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation for Santhera's lead orphan drug candidate, idebenone, for the treatment of Duchenne Muscular Dystrophy (DMD). The rare pediatric disease designation supplements the orphan drug designation granted by the FDA for idebenone to treat DMD in February 2007.

"We are pleased that the FDA has granted our request to designate idebenone for the treatment of DMD as a drug for a rare pediatric disease," said Thomas Meier, PhD, Chief Executive Officer of Santhera. "We already have Fast Track designation for idebenone in DMD and, on this basis, will be requesting priority review when we file the planned NDA. The potential to obtain a Rare Pediatric Disease Priority Review Voucher from the FDA, which we could retain for a future clinical development program of our own or sell, could provide additional value for the company in the future."

About Rare Pediatric Disease Designation
The FDA defines a "rare pediatric disease" as a disease that affects fewer than 200,000 individuals in the U.S. primarily aged from birth to 18 years. Under the FDA's Rare Pediatric Disease Priority Review Voucher program, a sponsor who receives an approval of a new drug application (NDA) or biologics license application (BLA) for a rare pediatric disease may be eligible for a voucher which can be redeemed to obtain priority review for a subsequent marketing application for a different product. The Priority Review Voucher may be sold or transferred an unlimited number of times.

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 Dystrophy (CMD), all areas of high unmet medical need. In June 2015, the Committee for Medicinal Products for Human Use (CHMP) recommended granting a marketing authorization in Europe for Raxone® for the treatment of LHON. 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@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.

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News release RPD designation



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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