FDA posts Zavesca briefing documents

FDA posted briefing documents ahead of Tuesday's meeting of the Endocrinologic and Metabolic Drugs Advisory Committee to discuss an sNDA from Actelion Ltd. (SIX:ATLN) for Zavesca miglustat to treat progressive neurological manifestations in patients with Niemann-Pick type C disease, a lysosomal storage disease. FDA said the key issue is whether the totality of the submitted data constitutes sufficient evidence to show a clinically meaningful effect for Zavesca. The agency noted that the Phase III Study 007 trial of Zavesca missed the primary endpoint of changes in horizontal saccadic eye movement velocity vs. no treatment. However, FDA said submitted data also included extensive assessments of other endpoints, as well as data from an extension study and retrospective studies that analyzed patients treated with Zavesca outside of clinical trials. ...