Today Santhera Pharmaceuticals announced the discontinuation of their clinical program with idebenone, a molecule under investigation for people living with Duchenne experiencing respiratory decline and receiving corticosteroids.
Data from their interim analysis concluded the study is unlikely to meet its primary endpoint. As a result, Santhera has decided to discontinue the program and withdraw its European marketing authorization application. However, the company remains focused on bringing its newly acquired drug candidate, varmorolone, to Duchenne patients as soon as possible.
This is very disappointing for our Duchenne community and all the families participating in the idebenone clinical trial program. We thank them for their commitment and contribution to understanding the risks and benefits of this drug candidate. If you participated in the SIDEROS study, you will need to stop taking idebenone treatment immediately and contact your study site for instruction if you haven’t already been contacted. Of note, the safety data has been reviewed throughout the conduct of the study and no safety concerns were detected.
We thank Santhera for their continued commitment to our Duchenne community. We look forward to the 6-month report on vamorolone and the VISION-DMD study expected in the second quarter of next year.
Santhera Pharmaceuticals is a Swiss specialty pharmaceutical company committed to developing and commercializing innovative medicines to meet the needs of patients living with rare and other diseases with high unmet medical needs.