Santhera plans early SNT-MC17/idebenone filing

Santhera Pharmaceuticals, a Swiss specialty company with a focus on neuromuscular diseases, plans to file, ahead of schedule, the Marketing Authorization Approval for its lead compound SNT-MC17/idebenone in Friedreich's Ataxia (FRDA) in Europe in the summer of this year. This decision follows discussions with representatives from regulatory authorities of several European Union member states, emphasizing the positive data from the company's collaborative clinical trial with the US National Institutes of Health. The results of this study showed improvement of neurological parameters and activities of daily living scores in FRDA patients after treatment with intermediate and high doses of SNT-MC17/idebenone for six months. The application for marketing authorization based on these positive data is fully supported by Takeda, Santhera's European marketing partner for SNT-MC17/idebenone for FRDA.

Santhera says that its early filing strategy takes advantage of new EU regulatory guidelines covering drug development for small patient populations. These guidelines aim to address patient needs by facilitating the approval of product candidates developed to treat diseases affecting only a small number of patients in the EU.