Santhera Receives Negative Opinion from the CHMP for its Marketing Authorization Application for Raxone® in DMD and Intends to Appeal this Opinion

Santhera Pharmaceuticals Holding AG / Santhera Receives Negative Opinion from the CHMP for its Marketing Authorization Application for Raxone® in DMD and Intends to Appeal this Opinion . Processed and transmitted by Nasdaq Corporate Solutions. The issuer is solely responsible for the content of this announcement.

Liestal, Switzerland, September 15, 2017 - Santhera Pharmaceuticals (SIX: SANN) announces that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a negative opinion for its Type II extension application for Raxone^® (idebenone) in Duchenne muscular dystrophy (DMD). Santhera plans to appeal the opinion and will seek a re-examination by the CHMP.

Santhera gave its oral explanation to the CHMP on Wednesday this week to further support the clinical relevance of the existing data in the proposed indication. The CHMP expressed uncertainties whether the phase III DELOS trial provides sufficient evidence of efficacy to allow a Type II variation of Santhera's existing marketing authorization for Raxone.

"We are surprised and disappointed by the opinion of the CHMP. Data from the phase III DELOS trial demonstrated statistically significant and clinically relevant evidence that Raxone slows the decline of respiratory function, and reduces the risk of bronchopulmonary complications and hospitalization in patients with DMD not using glucocorticoids," said Thomas Meier, PhD, CEO of Santhera. "These patients in the respiratory decline stage currently have no treatment options, and because we are confident that they could benefit from treatment with Raxone, we plan to appeal this opinion and seek re-examination."

The application was filed as a Type II Variation of the existing marketing authorization for Leber's hereditary optic neuropathy (LHON), and is based on data from Santhera's phase II (DELPHI) study and the successful pivotal phase III (DELOS) study, the latter in patients not taking concomitant glucocorticoids. The outcomes of the phase III DELOS study were published in several peer-reviewed journals: Buyse et al., The Lancet 2015, 385:1748-1757; McDonald et al., Neuromuscular Disorders 2016, 26:473-480, Buyse et al., Pediatric Pulmonology 2017, 52:580-515 and Mayer et al., Journal of Neuromuscular Diseases 2017, 4: 189-198.