CHMP adopts negative opinion for its Type II extension application for Raxone (idebenone) in Duchenne muscular dystrophy .- Santhera Pharmaceuticals
Santhera Pharmaceuticals) announces that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a negative opinion for its Type II extension application for Raxone (idebenone) in Duchenne muscular dystrophy (DMD). Santhera plans to appeal the opinion and will seek a re-examination by the CHMP. Santhera gave its oral explanation to the CHMP to further support the clinical relevance of the existing data in the proposed indication. The CHMP expressed uncertainties whether the phase III DELOS trial provides sufficient evidence of efficacy to allow a Type II variation of Santhera's existing marketing authorization for Raxone.
The application was filed as a Type II Variation of the existing marketing authorization for Leber's hereditary optic neuropathy (LHON), and is based on data from Santhera's phase II (DELPHI) study and the successful pivotal phase III (DELOS) study, the latter in patients not taking concomitant glucocorticoids. The outcomes of the phase III DELOS study were published in several peer-reviewed journals: Buyse et al., The Lancet 2015, 385:1748-1757; McDonald et al., Neuromuscular Disorders 2016, 26:473-480, Buyse et al., Pediatric Pulmonology 2017, 52:580-515 and Mayer et al., Journal of Neuromuscular Diseases 2017, 4: 189-198.
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