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CHMP rebuffs Santhera's Raxone for Leber's

January 19, 2013 1:08 AM UTC

EMA's CHMP issued negative opinions on Friday, including a recommendation against approval of an MAA from Santhera Pharmaceuticals Holding AG (SIX:SANN) for Raxone idebenone to treat Leber's hereditary optic neuropathy (LHON). The committee said the benefits of the ubiquinone analog did not outweigh its risks, noting that it did not consider the improvement in vision of three letters vs. placebo in LHON patients whose symptoms started in the previous five years to be "significant." CHMP also said that the reliability of results in a smaller group of LHON patients whose symptoms started in the previous year was "questionable." This smaller group showed an improvement in vision of 17 letters vs. placebo. Santhera had proposed restricting the use of Raxone to LHON patients whose symptoms started in the previous year. ...