Santhera Pharmaceuticals announces that the European Medicines Agency (EMA) has validated its marketing authorization application (MAA) for Puldysa® (idebenone) in the treatment of respiratory dysfunction in patients with Duchenne muscular dystrophy (DMD) who are not using glucocorticoids.
Validation confirms that the submission, which was filed as conditional marketing authorization (CMA), is complete and that the review process by the EMA’s Committee for Medicinal Products for Human Use (CHMP) has begun. Santhera expects an opinion by the CHMP around mid 2020.
“The initiation of the CHMP review of our application is an important milestone for patients with DMD who currently have no alternative treatment for the preservation of respiratory function. We are looking forward to working closely with the rapporteurs and CHMP during the review process to make Puldysa available to patients as soon as possible,”
said Kristina Sjöblom Nygren, MD, Chief Medical Officer and Head of Development at Santhera.
The MAA review follows the centralized procedure. If approved by the EMA, Puldysa will receive marketing authorization in all member states of the European Union, as well as in Norway, Liechtenstein and Iceland. The EMA has granted orphan drug designation for idebenone in DMD.