FDA Approves Orphalan’s Cuvrior for Adults with Wilson’s Disease

The U.S. Food and Drug Administration approved Cuvrior 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 United States with about one third of patients developing intolerance.

Photo: Mary Graper, vice president of scientific affairs for the Wilson Disease Association

Wilson’s disease is a rare inherited disorder of copper transport primarily affecting the liver and brain.

Trientine tetrahydrochloride is an oral trientine formulation. In the United States, trientine tetrahydrochloride has been granted with Orphan Drug designation for the treatment of Wilson’s disease excluding patients intolerant of penicillamine. It has been approved under the 505(b)(2) pathway for the treatment of adult stable Wilson’s disease patients who are successfully de-coppered and tolerant to penicillamine. The 505(b)(2) regulatory pathway is a type of New Drug application.

Orphalan commercializes its trientine tetrahydrochloride product in Europe under the name of Cuprior and expects to launch Cuvrior, which has been granted Orphan Drug designation by the FDA, in the United States by early 2023. The approval follows the New Drug application submission for the company’s product last year.

Orphalan recently completed a global phase 3 trial, CHELATE, which met its primary efficacy endpoint by demonstrating that Cuvrior was non-inferior to penicillamine as measured by non-ceruloplasmin copper. In consultation with the FDA, an assay based on total serum copper protein speciation was used for measuring this primary efficacy endpoint.

“Wilson’s disease is a devastating disorder affecting patients worldwide and for which there has remained a significant need for innovative new treatments,” said Mary Graper, vice president of scientific affairs for the Wilson Disease Association. “The approval of Orphalan’s Cuvrior is extremely promising and is reflective of Orphalan’s patient-driven approach. This marks the culmination of many years of work and is an important moment, offering new hope for patients affected by this disease.”

Author: Rare Daily Staff