Orphalan gets FDA approval for Cuvrior to treat Wilson's disease

France-based orphan drug developer Orphalan has received approval of Cuvrior, a new salt of trientine (trientine tetrahydrochloride) by the US Food and Drug Administration.

Cuvrior is approved for the treatment of adult patients with stable Wilson’s disease who are de-coppered and tolerant to penicillamine. Penicillamine is currently approved as a first-line treatment of Wilson’s disease in the USA with about one third of patients developing intolerance.

Orphalan recently completed a global phase III trial, CHELATE, which met its primary efficacy endpoint by demonstrating that Cuvrior was non-inferior to penicillamine as measured by non-ceruloplasmin copper (NCC). In consultation with the FDA, an assay based on total serum copper protein speciation was used for measuring this primary efficacy endpoint.