Santhera Pharmaceuticals has submitted a Marketing Authorisation Application (MAA) to the European Medicines Agency (EMA) for Puldysa (idebenone) in duchenne muscular dystrophy.
The company is seeking conditional marketing authorisation (CMA) for patients with DMD who are not using glucocorticoids, and the MAA is supported by data from a Phase II (DELPHI) study, the long-term DELPHI-Extension study, the pivotal Phase III (DELOS) study 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 Puldysa for up to six years.
“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, chief medical officer and head of development at Santhera.
The company has also announced plans to submit a new drug application (NDA) with the US Food and Drug Administration (FDA) following the completion of the currently ongoing double-blind, placebo-controlled Phase III (SIDEROS) study.