FDA Approved: Cuvrior for Wilson Disease

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). 

According to FiercePharma, the FDA approved Cuvrior in early May 2022. The therapy, developed by rare disease pharmaceutical company Orphalan, has already been approved in Europe as Cuprior

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
  • Fatigue
  • 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
  • Hepatitis

Learn more about Wilson disease

Jessica Lynn

Jessica Lynn

Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.

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