FDA approves Cuvrior for treatment of adults with stable Wilson’s disease

Orphalan announced FDA approval of Cuvrior for the treatment of adults with stable Wilson’s disease who are de-coppered and tolerant to penicillamine, according to a company press release.

Cuvrior (trientine tetrahydrochloride, Orphalan) recently demonstrated noninferiority to penicillamine by measurement of non-ceruloplasmin copper in a global, phase 3 trial; penicillamine is currently approved as a first-line treatment for Wilson’s with approximately one-third of patients developing intolerance.

“As a physician, I have seen firsthand how Wilson’s disease impacts the lives of patients and, until now, there have been few effective long-term treatment options available. The approval of Orphalan’s Cuvrior by the FDA is backed by positive data from Orphalan’s multicenter, multinational CHELATE trial — the first head-to-head controlled study of a new trientine salt vs. penicillamine,” Michael Schilsky, MD, professor and director of the Center for Excellence for Wilson Disease at Yale University, said in the press release. “For patients in need, Cuvrior represents a well-tolerated and effective alternative to penicillamine, the current standard of care.”

Trientine tetrahydrochloride, which is currently available in Europe as Cuprior, has been granted orphan drug designation by the FDA and is expected to launch in the U.S. by early 2023.