FDA Approves New Drug for Wilson's Disease

Megan Brooks

May 03, 2022

The US Food and Drug Administration (FDA) has approved oral trientine tetrahydrochloride (Cuvrior, Orphalan) for the treatment of adults with stable Wilson's disease who are decoppered and tolerant to penicillamine, the company has announced.

Wilson's disease is a rare genetic storage disease caused by a defect in a copper transporter gene, leading to copper accumulation in the liver, brain, eye, and peripheral nerves.

Trientine is a copper-chelating agent that removes copper from the body by forming a stable complex that is eliminated through urine. Trientine may also inhibit copper absorption from the intestinal tract.

In the global phase 3 CHELATE trial, Cuvrior met its primary efficacy endpoint by demonstrating noninferiority to penicillamine as measured by nonceruloplasmin copper.

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, the company said.

Trientine tetrahydrochloride is already on the market in Europe, where it is sold as Cuprior.

Orphalan expects to launch Cuvrior in the United States by early 2023. The drug has been granted orphan drug designation by the FDA.

Cuvrior is the first drug approved for Wilson's disease in the United States in more than 5 decades, according to Fierce Pharma.

"As a physician, I have seen first-hand how Wilson's disease impacts the lives of patients and, until now, there have been few effective long-term treatment options available," Michael Schilsky, MD, director, Center of Excellence for Wilson Disease, Yale University, New Haven, Connecticut, said in a statement.

"For patients in need, Cuvrior represents a well-tolerated and effective alternative to penicillamine, the current standard of care," Schilsky said.

"Wilson's disease is a devastating disorder affecting patients worldwide and for which there has remained a significant need for innovative new treatments," added Mary L. Graper, vice president of scientific affairs, Wilson Disease Association.

"This marks the culmination of many years of work and is an important moment, offering new hope for patients affected by this disease," Graper said.

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