EMA validates MAA for Raxone (idebenone) for Duchenne muscular dystrophy. - Santhera Pharma

Santhera Pharmaceuticals has announced that the European Medicines Agency (EMA) has validated its Marketing Authorization Application (MAA) for Raxone (idebenone) for Duchenne muscular dystrophy (DMD) in patients with respiratory function decline who are not taking concomitant glucocorticoids. Validation confirms that the submission, which was filed as Type II variation of the Company�s existing marketing authorization for Raxone, is complete and that the review process by the CHMP (Committee or Medicinal Products for Human Use) has begun. Santhera expects an opinion from the CHMP in the first quarter of 2017.

The regulatory dossier was submitted as a Type II Variation of the existing marketing authorization and is based on data from Santhera�s phase II (DELPHI) program and the successful pivotal phase III (DELOS) study which enrolled patients not taking concomitant glucocorticoids. These data, which demonstrated a statistically significant and clinically relevant benefit of Raxone treatment in preserving respiratory function compared to placebo, have been substantiated by a natural history study showing that the benefit observed in the Raxone-treated group would not have been expected from the natural course of the disease.