Santhera Applies for Euro Marketing Authorization for Duchenne Med

Santhera Pharmaceuticals (SIX: SANN) announces that it has submitted a marketing authorization application (MAA) for Puldysa® (idebenone) for the treatment of respiratory dysfunction in patients with Duchenne muscular dystrophy (DMD) to the European Medicines Agency (EMA). Santhera is seeking conditional marketing authorization (CMA).

The indication for Puldysa sought under CMA is the treatment of respiratory dysfunction in patients with DMD who are not using glucocorticoids. The MAA is supported by data from Santhera’s Phase II (DELPHI) study, the long-term DELPHI-Extension study, the pivotal Phase III (DELOS) study [1-5] and the recently completed SYROS study, a collection of long-term data from patients who completed the DELOS study and continued to be treated with idebenone for up to six years [6].

“The new data included in this regulatory submission confirm clinically relevant patient benefits and long-term therapeutic efficacy with idebenone in patients with DMD. Thereby, we have closed earlier data gaps and respond to requirements from the regulatory authorities,” said Kristina Sjöblom Nygren, MD, Chief Medical Officer and Head of Development at Santhera.

In line with previous communications, Santhera plans to submit a new drug application (NDA) with the US FDA following the completion of the currently ongoing double-blind, placebo-controlled Phase III (SIDEROS) study that investigates the efficacy and safety of idebenone in delaying the loss of respiratory function in patients with DMD taking glucocorticoid steroids over a period of 18 months (ClinicalTrials.gov Identifier: NCT02814019; www.siderosdmd.com). With 266 patients to be enrolled, SIDEROS is the largest actively recruiting clinical trial in DMD. To date, the study has 214 patients enrolled at approximately 60 clinical trial sites in Europe, the US and Israel, with patient recruitment expected to complete in 4Q 2019. Eligible patients who complete SIDEROS are offered to enroll in the open-label SIDEROS-Extension study (ClinicalTrials.gov Identifier: NCT03603288).

Idebenone for the treatment of DMD has been granted orphan drug designation by European, US, Swiss and Australian authorities, and it has fast track designation in the US.

https://www.globenewswire.com/news-release/2019/05/27/1850103/0/en/Santhera-Submits-Marketing-Authorization-Application-to-the-European-Medicines-Agency-for-Puldysa-Idebenone-in-Duchenne-Muscular-Dystrophy.html