Earlier this month the FDA approved an ORAL specialty therapy, Cuvrior (trientine tetrahydrochloride) from Orphalan SA, for the treatment of adults with stable Wilson’s disease who are decoppered and tolerant to penicillamine. Wilson’s disease is a devastating disorder affecting patients worldwide. It 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. It is the first drug approved for Wilson’s disease in the United States in more than 5 decades.
Approximately one in 90 people may be carriers of the disease gene. Current prevalence is estimated to be only about 2,000-3,000 cases in the United States. Other affected individuals have been misdiagnosed with other neurological, liver, or psychiatric disorders.
The big question is….. “Is this a new drug??”
In fact, trientine tetrahydrochloride was first approved by the FDA is 1985….. Yes…. 37 years ago!
As we are seeing, there has been an increasing number of ‘old’ therapies being reapproved due to ‘tweaks’ in formulations or reissuing in a different form (usually oral).
Orphalan announced that pricing will be released near to product launch.
Orphalan expects to launch Cuvrior in the United States by early 2023.
Given the very small patient population in the US it is a sure bet that Cuvrior will launch through limited distribution.
FDA Approves New Drug for Wilson’s Disease
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 trials 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.