Posted by Michael Wonder on 14 Jul 2016
Santhera updates on U.S. regulatory filing for Raxone (idebenone) in Duchenne muscular dystrophy
14 July 2016 - Santhera Pharmaceuticals announces that it has received written correspondence from the U.S. FDA on its proposed subpart H approval pathway for Raxone in Duchenne muscular dystrophy patients not taking concomitant glucocorticoids.
Santhera had proposed that the planned SIDEROS trial would provide confirmatory evidence of efficacy in these patients whilst expanding the label to include the treatment of glucocorticoid-using patients.
From its review of this strategy, the FDA concluded that results from the SIDEROS trial, which is powered to detect a difference in the established surrogate endpoint forced vital capacity percent predicted in glucocorticoid-using patients, should be provided at the time of filing to support an NDA for the treatment of DMD patients irrespective of their glucocorticoid use status.
