www.fdanews.com/articles/207646-fda-okays-new-treatment-option-for-patients-with-wilsons-disease
FDA Okays New Treatment Option for Patients With Wilson’s Disease
May 3, 2022
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.