Raxone idebenone regulatory update

Santhera said EMA accepted for review a resubmitted MAA for Raxone idebenone to treat Leber's hereditary optic neuropathy (LHON). The company expects a decision in 1H15. The company withdrew an MAA for Raxone in March 2013 after EMA's CHMP said the benefits of the compound did not outweigh its risks. The resubmitted MAA includes new efficacy data from an ongoing expanded access program for the ubiquinone analog as well as natural history data collected in collaboration with the European Vision Institute Clinical Research Network.

In January, the French National Agency for Medicines and Health Products Safety (ANSM) granted an Authorization of Temporary Use (ATU) for Raxone which allows patients in France to receive Raxone before EU approval. Santhera said the data package submitted to ANSM is comparable to the submitted MAA dossier. ...