The European Medicines Agency (EMA) has announced the validation of Santhera’s Marketing Authorisation Application for Puldysa (idebenone) in Duchenne muscular dystrophy (DMD).
The governing body has begun to review the application for the drug as a treatment for respiratory dysfunction in patients with DMD who are not using glucocorticoids.
The validation confirms that the submission, which was filed as conditional marketing authorisation, 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.
If approved by the EMA, Puldysa will receive marketing authorisation in all member states of the European Union, as well as in Norway, Liechtenstein and Iceland.
“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,” said Kristina Sjöblom Nygren, chief medical officer and head of development at Santhera.
She continued, “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.”
The news comes hot on the heels of The Medicines and Healthcare Products Regulatory Agency (MHRA) renewing its Early Access to Medicines Scheme (EAMS) scientific opinion for the drug for a further year.
DMD is one of the most common and devastating types of progressive muscle weakness and degeneration starting at an early age and leading to early morbidity and mortality due to respiratory failure. It is a genetic, degenerative disease that occurs almost exclusively in males with an incidence of up to 1 in 3,500 live male births worldwide.