Santhera Pharmaceuticals said on Friday that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) gave 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 on Wednesday this week to 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.
Thomas Meier, CEO of Santhera, said he was “surprised and disappointed by the opinion of the CHMP.” He said that the data from the phase III DELOS trial showed statistically significant and clinically relevant evidence that Raxone slows the decline of respiratory function, and reduces the risk of bronchopulmonary complications and hospitalization in patients with DMD not using glucocorticoids
“These patients in the respiratory decline stage currently have no treatment options, and because we are confident that they could benefit from treatment with Raxone, we plan to appeal this opinion and seek re-examination,” he said.
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.
Raxone should slow the decline of respiratory function in patients with DMD who are currently not taking glucocorticoids. The indication would include patients who were previously treated with glucocorticoids or in whom glucocorticoid treatment is not tolerated or is considered inadvisable.
Image: Bottles containing 180 tablets of Raxone (idebenone 150 mg). Source: Santhera