The company focuses on the development and commercialization of innovative medicines for rare neuromuscular and pulmonary diseases with high unmet medical need, including vamorolone, a first-in-class dissociative steroid, and lonodelestat (POL6014) for the treatment of cystic fibrosis. Santhera has also out-licensed the rights to its first approved product, Raxone®, for the treatment of Leber's hereditary optic neuropathy, to Chiesi Group.