Santhera publishes pivotal trial results of idebenone and goes for EU approval
Liestal/Newscastle – Swiss Santhera Pharmaceuticals and researchers from Newcastle University have published results from a pivotal clinical trial demonstrating that the firm’s synthetic coenzyme Q10 derivative idebenone significantly improves vision and perception of colour in a subgroup of patients with the rare mitochondrial disease Leber's Hereditary Optic Neuropathy (LHON). The 10,000 to 20,000 patients in Europe and the US, which suffer from the maternally inherited condition, initially lose the sight in one eye due to a malfunction of complex I of the mitochondrial respiratory chain and subsequent retinal apoptosis. Typically, they lose the sight in their other eye within the next 3 to 6 months (so called discordant visual acuity). Researchers from Newcastle, Munich, and Montreal now demonstrated for the first time in 85 patients, that treatment with a 900mg dose of idebenone over two years could significantly improve visual acuity in a patient subgroup carrying the G11778A or G3460A mitochondrial DNA mutations, which are found in 80% of all LHON patients.
The Newscastle group led by Professor Patrick Chinnery reported that idebenone treatment resulted in a marked improvement of vision in severely affected patients, who were not able to read any letter on a chart. In nine blind patients (12 eyes) out of 36 patients (61 eyes) taking idebenone, vision improved to an extent that patients were able to read at least one row of letters on the chart (20%). In contrast not a single patient of the 26 who were taking the placebo improved to that extent.
The results published in yesterday’s issue of BRAIN are part of a data package accepted by the EU drug authority on July 22th which now reviews the firm’s MAA in this indication. Market approval in LHON could be expected at the end of 2012 earliest, according to the Swiss specialist for neuromuscular diseases. The drug will be available trough a Named Patient Programme until the EMA has come to a decision. The greatest improvement was seen in patients who had deteriorated in one eye more than the other. The RHODOS study does not deliver data from patients who have been affected from the disease for more than 5 years. Idebenone has already gotten market approval in Canada as treatment for Friedreichs ataxia but was rejected the the EMA in this indication.