Health Care Professionals


Santhera is passionate about medical science and about developing new medical treatments for patients with rare and other diseases. Our focus is on the development of therapies for rare neuromuscular and pulmonary conditions with high unmet medical needs.

Our pipeline

Santhera has an exclusive license for all indications worldwide to vamorolone, a first-in-class dissociative steroid with novel mode of action, currently investigated in a pivotal study in patients with DMD as an alternative to standard corticosteroids. The clinical stage pipeline also includes lonodelestat (POL6014) to treat cystic fibrosis (CF) and other neutrophilic pulmonary diseases and an exploratory gene therapy approach targeting congenital muscular dystrophies.

Santhera out-licensed ex-North American rights to its first approved product, Raxone® (idebenone), for the treatment of Leber's hereditary optic neuropathy (LHON) to Chiesi Group.