Orphalan

Orphalan SA is a global, commercial-stage orphan drug company focused on rare diseases, with a primary anchor in Wilson disease. The company has successfully developed and gained marketing authorization for its lead product, trientine tetrahydrochloride (Cuprior®/Cuvrior®), which is now available in over 30 countries. Through recent strategic acquisitions, such as Orphelia Pharma, and expansion into key markets like China and the US, Orphalan is building a broader rare disease portfolio, particularly in pediatric neurology and oncology. The company employs over 100 people and maintains a dedicated international team across more than 20 countries.

Strategic development and commercialization platform for orphan drugs, focusing on identifying and advancing existing molecules with improved formulations through targeted regulatory pathways for rare diseases.

In Wilson disease, Orphalan's trientine competes primarily with older chelating agents like penicillamine and generic trientine dihydrochloride, against which it positions itself as a modern, stable formulation. The broader rare disease landscape is fragmented with many small biotechs and specialty pharma companies. Orphalan's strategy of acquisition and global commercialization aims to build scale and compete effectively in this niche but growing sector.