Santhera re-files for EU approval of Raxone in the treatment of LHON

Swiss drugmaker Santhera Pharmaceuticals (SIX: SANN) has re-filed its Marketing Authorization Application for Raxone (idebenone) in the treatment of Leber's hereditary optic neuropathy (LHON) with the European Medicines Agency.

An earlier application was withdrawn in March 2013 in order to include additional clinical efficacy data. The compound has been granted orphan drug designation in the European Union. Raxone would become the first product authorized for the treatment of this rare, inherited disease which otherwise invariably leads to blindness. Santhera says it expects a decision from the EMA in the first half of 2015.

Santhera discussed the additional clinical efficacy data and the overall content of the revised MAA dossier with several EU member states prior to proceeding with the re-filing. Earlier this year the French National Agency for the Safety of Medicine and Health Products (ANSM) granted a temporary authorization for use for Raxone in LHON patients in France based on a data package comparable to the submitted MAA dossier (The Pharma Letter January 21).

Optimistic on MAA approval; plans discussions with US FDA

"We are optimistic that the new data on Raxone which we have submitted in this MAA dossier will address the issues raised by the CHMP during the previous procedure," commented Thomas Meier, chief executive of Santhera, adding: "The totality of data included in the re-submission provides compelling evidence that Raxone can protect against vision loss in the early stages of the disease and promote recovery of vision already lost."

Santhera’s drug has previously been granted an US orphan drug designation for the treatment of LHON and the company will now approach the US Food and Drug Administration for discussions on a regulatory path to a US approval based on the data package filed in the European MAA.