GMP-Orphan

Orphalan (formerly GMP-Orphan) is a specialty pharmaceutical company based in Paris, France, dedicated to developing and commercializing treatments for rare (orphan) diseases. Founded in 2011, the company's flagship product is Cuprior (trientine tetrahydrochloride), a next-generation copper chelator for Wilson's disease that offers improved stability and room-temperature storage. Cuprior is approved in the European Union, the United States (as Cuvrior), and China, marking a significant advancement in the management of this rare genetic disorder. In 2025, Orphalan expanded its portfolio by acquiring Orphelia Pharma, adding pediatric treatments such as Kigabeq for infantile spasms and Ivozall for acute lymphoblastic leukemia to its commercial offerings. The company operates with a patient-centered approach, collaborating with academic institutions and healthcare professionals to address unmet medical needs in rare metabolic, neurological, and oncological conditions.