Pharmaprojects R&D Pipeline News Feed
First ever approval for Freidreich's Ataxia therapeutic
19 Aug 2008
Santhera Pharmaceuticals has received the first marketing authorisation in the world for a Freidreich's Ataxia therapy, following a conditional approval by Health Canada for Catena (ibedenone). Launch is expected in at the end of October. The product is also under review by health authorities in the EU and Switzerland.
Catena demonstrated significant efficacy and clinically relevant improvements in Friedrich's Ataxia patients; however, Santhera also agreed to submit additional data from an ongoing US Phase III trial in around 60 patients to confirm efficacy. It is also in Phase II trials for Duchenne muscular dystrophy and Leber's hereditary optic neuropathy, and has European and US orphan drug status for all three indications.
Ibedenone was previously developed and launched by Takeda for improving cerebral energy metabolism and treating sequelae of cerebral apoplexy, such as stroke. It is a small molecule optimised to facilitate the balance and flow of electrons within mitochondria. Through preserving mitochondrial function and protecting cells from oxidative stress, it is thought to prevent cell damage and increase energy production within impaired nerve and muscle tissue.
