Santhera Pharmaceuticals : Raxone® Receives First Positive EAMS Scientific Opinion from UK’s MHRA in Duchenne Muscular Dystrophy

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The aim of the EAMS is to provide patients with life threatening or seriously debilitating conditions access to medicines that do not yet have a marketing authorization when there is a clear unmet medical need.1 The MHRA decision allows patients with DMD, who meet criteria defined under this scheme, to gain access to Raxone, an investigational medicinal product currently under review for DMD for Marketing Authorization by the European Medicines Agency (EMA).

Under the EAMS, and as shown in the public assessment report,2 Raxone is indicated for slowing the decline of respiratory function in patients with DMD from the age of 10 years who are currently not taking glucocorticoids. The decline of respiratory function must be confirmed by repeated measure- ments prior to initiation of treatment. Raxone can be used in patients previously treated with gluco- corticoids or in patients in whom glucocorticoid treatment is not tolerated or is considered inadvisable.

"We're proud to receive the positive EAMS scientific opinion for Raxone in the UK and to have our lead compound designated as a promising innovative medicine, the first for a drug intended for the treatment of DMD," said Thomas Meier, PhD, CEO of Santhera. "This decision allows patients with DMD to receive treatment for respiratory function decline who otherwise would not have access to such treatment options."

"This is excellent news for patients with respiratory decline in Duchenne muscular dystrophy," said Janet Bloor, Chair of the Board of Trustees at Action Duchenne. "The need for new treatments in DMD is very great and the EAMS can help to accelerate access for patients. Action Duchenne was pleased to provide advice during the development of the EAMS program and we are delighted to see this first positive opinion in DMD."

The UK's industry-sponsored EAMS aims to give patients with life threatening or seriously debilitating conditions access to medicines that do not yet have a marketing authorization when there is a clear unmet medical need. Under the scheme, the MHRA provides a scientific opinion on the benefit/risk balance of the medicine, based on the data available when the EAMS submission was made. The opinion lasts for a year and can be renewed. The scheme is voluntary and the opinion from MHRA does not replace the normal licensing procedures for medicines.

Santhera Pharmaceuticals (SIX: SANN) is a Swiss specialty pharmaceutical company focused on the development and commercialization of innovative pharmaceutical products for the treatment of or- phan mitochondrial and neuromuscular diseases. Santhera's lead product Raxone® (idebenone) is authorized in the European Union, Norway, Iceland and Liechtenstein for the treatment of Leber's hereditary optic neuropathy (LHON). For Duchenne muscular dystrophy (DMD), Santhera has filed a Marketing Authorization Application in the European Union and Switzerland for DMD patients with respiratory function decline who are not taking glucocorticoids. In collaboration with the U.S. National Institute of Neurological Disorders and Stroke (NINDS) Santhera is developing Raxone® in a third indication, primary progressive multiple sclerosis (PPMS), and omigapil for congenital muscular dys- trophy (CMD), all areas of high unmet medical need. For further information, please visit the Company's website www.santhera.com.

Raxone® is a trademark of Santhera Pharmaceuticals.

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

Sue Schneidhorst, Head Group Communications Phone +41 61 906 89 26

sue.schneidhorst@santhera.com

Hans Vitzthum, LifeSci Advisors, LLC Chris Welsh, Consilium Strategic Communications Phone +1 212 915 2568 Phone +1 646 586 2113

hans@lifesciadvisors.com santhera@consilium-comms.com

Amber Fennell, Consilium Strategic Communications Phone +44 20 3709 5700

santhera@consilium-comms.com

This communication does not constitute an offer or invitation to subscribe for or purchase any secu- rities of Santhera Pharmaceuticals Holding AG. This publication may contain certain forward-looking statements concerning the Company and its business. Such statements involve certain risks, uncer- tainties 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.

1 Medicines and Healthcare products Regulatory Agency Patient safety and Marketing authorisations, variations and licensing guidance. Available at: https://www.gov.uk/guidance/apply-for-the-early-ac- cess-to-medicines-scheme-eams (June 2017)

2 Public assessment report. Available at https://www.gov.uk/government/publications/early-access- to-medicines-scheme-eams-scientific-opinion-raxone-to-treat-the-decline-of-respiratory-function-in- patients-with-duchenne-muscular-dys

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