FDA Okays New Treatment Option for Patients With Wilson’s Disease

The FDA has approved Orphalan’s Cuvrior (trientine tetrahydrochloride) for the treatment of adult patients with Wilson’s disease, a rare inherited disorder that causes a buildup of copper in the liver, brain and other organs.

The approval is for patients who have been de-coppered and who are tolerant to penicillamine, the existing first-line treatment approved by the agency in 1951.

The U.S. approval was based on data from a global phase 3 study in which Cuvrior demonstrated noninferiority to penicillamine.

In the EU, Cuvrior — sold as Cuprior — received marketing authorization in September 2017. The French company plans to launch the drug in the U.S. in early 2023.