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Santhera's Raxone turned down for rare eye condition

EMA concerned about drug's ability to improve eyesight

European regulators have refused to recommend Santhera's Raxone be approved to treat the rare eye condition Leber's hereditary optic neuropathy (LHON).

The European Medicines Agency (EMA) said it had concerns about the drug's effectiveness, with trial data demonstrating that in LHON patients whose symptoms started in the previous five years, Raxone (idebenone) was no better than placebo in improving people's sight.

This measure was based on a patient's ability to read letters on a standard eye chart, with patients taking Raxone able to distinguish just three more letters than the placebo cohort.

Santhera had also requested the EMA consider restricting Raxone solely for use in patients with LHON whose symptoms had started in the previous year, where the improvement in eyesight was superior.

However, the EMA said it could not recommend the drug in this indication as there were only 28 patients in this sub-group of the trial – too small a number to rule out a spontaneous improvement.

German pharma company Santhera said it had decided to request a re-examination of the opinion, claiming that the agency's Committee for Medicinal Products for Human Use  (CHMP) had not fully considered the benefits of Raxone in patients for whom treatment was most urgent.

The company also wants to be able to supply any further clinical data that might be necessary on a post-approval basis, rather than to support an initial recommendation, according to Santhera's CEO Thomas Meier.

He said: “Since there were no concerns expressed about the safety of Raxone, and the observed treatment benefit on visual acuity in these patients was clinically relevant and consistent with the published literature, we believe that we should be allowed to address the remaining concerns through further confirmatory clinical work to be conducted post-approval.

“To this extent, we continue to strive for approval so that Raxone becomes available as the first treatment for patients with this devastating disease.”

LHON is a genetic disorder that typically affects young adults, leading to blindness within a few months of the first onset of symptoms.

21st January 2013

From: Sales



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