Orphalan Touts First FDA-Approved Wilson’s Disease Treatment Since 1970

Orphalan announced the U.S. Food and Drug Administration has granted full approval to Cuvrior (trientine tetrahydrochloride) for the treatment of Wilson’s Disease, a rare genetic disorder. The approval announcement marks the first new treatment available for patients in over five decades.

In patients with Wilson’s Disease, the body is prevented from removing extra copper, which causes the element to build up in the liver, brain, eyes and other organs. The disease is caused by the disruption of or mutations to the ATP7B gene, which plays an important role in the movement of excess copper from the liver to the bile that is eventually excreted from the body. Approximately one in 90 people may be carriers of the disease gene, and the disease is estimated to impact 9,000 people in the U.S.

Liver dysfunction is one of the hallmarks of the disease, and typical symptoms of liver dysfunction, like swelling of the legs and abdomen and jaundice, are among the first signs of Wilson’s Disease. One of the tell-tale signs of the disease is a Kayser-Fleischer ring, a brownish-yellow ring in the eye caused by excess copper on the inner surface of the cornea.

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