For the last 52 years, individuals with Wilson disease have been relying, largely, on the same treatment options. These options, designed to remove copper from the body, include Cuprimine (penicillamine). However, the treatment landscape for Wilson disease has now changed with the onset of a newly approved drug called Cuvrior (trientine tetrahydrochloride).
To begin, patients must take chelating agents to stop copper absorption. Next, Cuvrior is administered via 8-12 pills taken daily. It attaches to copper to form a compound which can then be excreted and removed from the body. The approval hinged on Orphalan’s proprietary copper test which helped to measure biomarkers and highlight the treatment’s efficacy.
Importantly, Cuvrior also offers some flexibility to patients. The treatment is stable at room temperature and provided in a convenient pack, which patients can take on the go. Cuvrior should be available for patients beginning in 2023.
About Wilson Disease
Wilson disease is a rare genetic disorder in which the body is unable to metabolize copper. As a result, excessive amounts of copper accumulate in the liver, brain, and other organs. ATP7B gene mutations cause Wilson disease. While copper begins to accumulate at birth, symptoms typically manifest around ages 12-13. Symptoms may appear, however, between ages 6 to 45. Without treatment, Wilson disease can be fatal. Symptoms may include:
- Kayser-Fleisher ring (a brown ring around the iris)
- Jaundice (yellowing of the skin, eyes, and mucous membranes)
- Abdominal pain and swelling
- Appetite loss
- Difficulty swallowing
- Depression or anxiety
- Easy bruising
- Low white blood cell count
- Fluid buildup in the legs or abdomen
- Difficulty walking
- Psychiatric difficulties
- Liver disease
Learn more about Wilson disease.