Santhera is building a Duchenne muscular dystrophy (DMD) product portfolio to treat patients irrespective of causative mutations, disease stage or age. A marketing authorization application for Puldysa® (idebenone) is currently under review by the European Medicines Agency. Santhera has an option to exclusively in-license vamorolone, a first-in-class anti-inflammatory drug candidate with novel mode of action, in all indications and all countries worldwide except Japan and South Korea. Vamorolone is currently investigated in a pivotal study in patients with DMD to replace standard corticosteroids. In February 2018, Santhera in-licensed from Polyphor a clinical stage selective inhibitor (lonodelestat, POL6014) of human neutrophil elastase with the potential to treat cystic fibrosis (CF) and other lung diseases. The pipeline also includes omigapil and an exploratory gene therapy approach targeting congenital muscular dystrophies.