Orphagen Pharmaceuticals does translational research, discovering new classes of small molecule drugs to promising but previously unexplored drug targets. Our goal is to deliver revolutionary drugs to treat diverse, chronic diseases in collaboration with partners. 

Orphagen has identified novel approaches to several orphan indications, including retinitis pigmentosa, Cushing’s syndrome and adrenocortical cancer. The pharmaceutical industry recognizes the advantages of commercializing an orphan drug because of shorter clinical development times and more generous reimbursement.