Wilson's disease treatment from Orphalan gets the go-ahead after PhIII
Cuvrior, the Orphalan treatment for adult patients with stable Wilson’s disease, has gotten approval from the FDA.
The OK comes after a Phase II trial dubbed CHELATE, which met primary efficacy endpoints by showing that Cuvrior was not inferior to penicillamine as measured by non-ceruloplasmin copper.
Wilson’s disease is a rare inherited disorder of copper transport that primarily affects the liver and brain. Most people diagnosed with the disease learn of it between the ages of 5 and 35, and it can delay the development of healthy nerves, bone, collagen and skin pigment melanin. It is an inherited disease, and can lead to cirrhosis, liver failure and kidney problems.
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