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Santhera stops Phase 3 SIDEROS Study and Development of Puldysa®

Today, Santhera announced the discontinuation of its Phase 3 SIDEROS study with Puldysa® (idebenone) in patients with Duchenne muscular dystrophy (DMD) who are in respiratory decline and receive concomitant glucocorticoid treatment. Data from an interim analysis conducted by the independent Data and Safety Monitoring Board (DSMB) concluded that the study was unlikely to meet its primary endpoint.

Click here to read the full press release

As a consequence, Santhera will discontinue the study, withdraw the European marketing authorization application and end the global development program for Puldysa. Santhera intends to initiate a restructuring plan for the business with a focus on retaining key functions for bringing DMD drug candidate vamorolone to patients and execute on its other pipeline programs.


We are hosting a webinar Tuesday October 20 (1pm New York, 6pm London) for anyone interested. The webinar will be recorded and made available for sharing afterwards. Next to a presentation by Santhera on the discontinuation of the SIDEROS study, it will give attendees the opportunity to ask questions. Please send any questions for the company in advance so we can prepare the webinar correctly.
RSVP required. Please register for this webinar by clicking the button below.
WDO + Santhera webinar on SIDEROS study stop