ORPHELIA Pharma is a French pharmaceutical company dedicated to the development and marketing of pediatric drugs for oncology and CNS diseases. ORPHELIA’s mission is to significantly improve essential medicines administered to children through the development of efficient, reliable and safe drugs with child-friendly formulations. We focus on developing optimized formulations for the pediatric population of approved drugs as well as obtaining the approval for existing drugs in new pediatric orphan indications. We are also leading R&D projects aiming at developing new drugs for pediatric orphan or ultra-rare indications. ORPHELIA’s portfolio encompasses drugs for the treatment of West Syndrome (epilepsy), relapsed or refractory high-risk neuroblastoma and acute lymphoblastic leukemia, with a first marketing authorization expected in 2018. ORPHELIA is headquartered in Paris with a secondary site in Lyon area. Key investors include initiative Octalfa (lead investor) and Pierre Fabre Medicament.