First Patients Enrolled in UK's Early Access to Medicines Scheme for Santhera's Raxone® in Duchenne Muscular Dystrophy (DMD)

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Liestal, Switzerland, September 11, 2017 - Santhera Pharmaceuticals (SIX: SANN) announces enrollment of first DMD patients with respiratory function decline not taking glucocorticoids into UK's Early Access to Medicines Scheme (EAMS) for Raxone^®.

In June, Raxone^® (idebenone) was granted a positive scientific opinion through the Early Access to Medicines Scheme (EAMS) by the UK's Medicines and Healthcare products Regulatory Agency (MHRA) - the first drug approved under the EAMS for patients with DMD. 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 Marketing Authorization in DMD by the European Medicines Agency (EMA). Enrollment into the EAMS documents the first use of Raxone outside of a clinical trial for patients with DMD.

"I am pleased to be able to offer Raxone to several of my patients in respiratory decline, as there are no other medical treatments available," said Dr. Dipansu Ghosh, a respiratory physician at a DMD center based in Leeds.

To date, 15 specialist DMD centers in the UK have received training under the requirements of the EAMS. Several additional sites have expressed an interest to be trained and are currently undergoing local approval processes.

"At Action Duchenne we were encouraged by the positive EAMS opinion, earlier this summer. Particularly, for young people living with Duchenne who have respiratory decline," said Janet Bloor, Chair of Trustees of Action Duchenne. "I am delighted that respiratory function is being considered by the regulatory agencies. This will hopefully pave the way for more potential treatments that may benefit the wider spectrum of DMD patients."

Under the EAMS, and as shown in the public assessment report, ¹ 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 measurements prior to initiation of treatment. Raxone can be used in patients previously treated with glucocorticoids or in patients in whom glucocorticoid treatment is not tolerated or is considered inadvisable.