The authorized regulators have acknowledged the choice of pathway for conditional marketing authorization.
Santhera Pharmaceuticals has announced its intention of filing an application with European Medicines Agency (EMA) for Conditional Marketing Authorization (CMA) for Puldysa® (idebenone) to treat respiratory dysfunction in Duchenne muscular dystrophy (DMD). Reportedly, completion and filing of the CMA is planned in the second quarter of 2019 for Puldysa after taking scientific advice from EU regulatory agencies.
Santhera has substantiated and expanded its preceding regulatory report by adding clinical data from patients treated with idebenone, addressing requests from regulatory authorities, new comprehensive natural history data and new analyses of data submitted previously. This new data entirely demonstrates patient benefits which are clinically relevant and sustained therapeutic efficacy of idebenone during treatment for up to six years in DMD patients.
According to the press release by Santhera, for Puldysa, initial indication is to treat respiratory dysfunction in DMD patients not using glucocorticoids. Santhera will file application on basis of its recently completed SYROS study, DELPHI Phase II study, the long-term DELPHI-Extension study and the crucial Phase III (DELOS) study [1-5].
The forthcoming filing will include clinical trial data demonstrating therapeutic potential of idebenone in treatment of respiratory dysfunction in DMD patients, potential of idebenone to stretch the time to milestones of disease progression which are clinically relevant, beneficial treatment effects of idebenone and sustained year on year for up to six years and support of idebenone in preserving respiratory function.
Kristina Sjöblom Nygren, Chief Medical Officer, MD and Head of Development, Santhera reportedly noted that team of Santhera worked relentlessly to assemble new data to strengthen regulatory report for Puldysa as a potential treatment for patients with DMD. The authorized regulators have acknowledged the choice of pathway for conditional marketing authorization and the company is in final preparations for submitting the filing dossier, Nygren further added.
Authorities from Europe, the U.S., Australia and Switzerland have granted orphan drug designation to idebenone for the treatment of DMD.