Santhera Files Marketing Authorization Application in the European Union for Raxone® for the Treatment of Duchenne Muscular Dystrophy

Santhera Pharmaceuticals Holding AG / Santhera Files Marketing Authorization Application in the European Union for Raxone® 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, May 31, 2016 - Santhera Pharmaceuticals (SIX: SANN) announces that it has submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for Raxone^® (idebenone) for the treatment of Duchenne muscular dystrophy (DMD) in patients with respiratory function decline and not taking concomitant glucocorticoids. The new indication was submitted as Type II variation of the company's existing marketing authorization for Raxone granted last year. Raxone has Orphan Drug Designation for DMD in the EU.

The regulatory dossier now submitted as Type II Variation of the existing marketing authorization summarizes data from Santhera's phase II (DELPHI) program and the successful pivotal phase III (DELOS) study which demonstrated a statistically significant and clinically relevant benefit of Raxone treatment in slowing the rate of respiratory function loss compared to placebo in patients with DMD not taking concomitant glucocorticoids. This benefit also translated to fewer bronchopulmonary events (e.g. airway infections), shorter event duration and less antibiotic treatment in the Raxone-treated patients. The data have been substantiated by a natural history study showing that the significant decline of respiratory function observed in the placebo group of DELOS reflected the natural course of the disease whilst the outcome for Raxone-treated patients did not. The MAA also includes data from a patient-centered benefit-risk survey which highlighted the importance of treating pulmonary disease in patients with DMD. The findings showing that Raxone slows the rate of respiratory function decline, decreases the proportion of patients crossing clinically relevant functional thresholds and increases the time until such thresholds are reached, represent an important treatment effect and are of major clinical relevance for patients with DMD. Raxone (900 mg/day) was safe and well tolerated with adverse event rates comparable to placebo.