Orphalan Cuvrior Earned Earlier Use With Head-To-Head Trial Against Frontline Wilson’s Disease Drug

French orphan specialist developed new assay for primary endpoint to show non-inferiority of its room temperature-stable salt of trientine, earning a broader FDA approval than generically available second-line trientine products.

In Wilson's disease, copper accumulates in tissues.
In Wilson's disease, copper accumulates in tissues. • Source: Nielsen Hobbs; the Pink Sheet | Shutterstock images

Orphalan’s Cuvrior (trientine tetrahydrochloride) will join the US market five years after European Union approval, but with an indication for an earlier line of therapy for patients with Wilson’s disease, a rare inherited disorder of copper transport.

The FDA approved Cuvrior, a new salt of the copper chelating agent trientine, on 28 April 2022 for treatment of adult patients with stable Wilson’s disease who are de-coppered and...

More from Rare Diseases

More from Pink Sheet