CHMP maintains negative opinion for Raxonea poposed treatmant for Duchenne muscular dystrophy.- Santhera.

Santhera Pharmaceuticals announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) maintained its negative opinion on the Type II extension application for Raxone (idebenone) in Duchenne muscular dystrophy (DMD) following a re-examination procedure.

The CHMP concluded that an approval for Raxone in DMD, applied as a Type II variation of the existing marketing authorization, cannot be granted at the present time based on the current existing evidence. Although the positive outcome of the Phase III DELOS trial was acknowledged, the CHMP has invited Santhera to present additional data to further link the observed treatment effects on respiratory function outcomes to patient benefit. Respiratory decline is one of the leading causes of death in patients with DMD and there is currently no approved treatment.

Santhera remains fully committed to addressing this unmet need and is convinced of the treatment benefits of Raxone in DMD. Santhera intends to collect further evidence to strengthen the clinical data package for Raxone in preparation of a refiling of a Marketing Authorization Application (MAA) in Europe.