Posted by Michael Wonder on 07 Oct 2020
Santhera to discontinue Phase 3 SIDEROS trial and development of Puldysa in Duchenne muscular dystrophy and focus on vamorolone
6 October 2020 - As a consequence, Santhera will discontinue the study, withdraw the European marketing authorization application and end the global development program for Puldysa.
Based on the now completed interim analysis which tested for efficacy, the DSMB has recommended the SIDEROS study be discontinued due to futility. The interim analysis was based on the primary endpoint of the study, the change of forced vital capacity % predicted from baseline to 18 months of treatment. The outcome revealed that the probability of reaching the primary endpoint at the end of the study is too small to merit the continuation of the study. There were no safety concerns noted by the DSMB.
