12 April 2018 | News
Wilson Therapeutics lead candidate has received Fast Track designation in the US and Orphan Drug Designation for the treatment of Wilson disease in the US and EU
M&A deal making continues with Alexion Pharmaceuticals swapping up Wilson Therapeutics AB for USD 855 million. Swedish drugmaker Wilson Therapeutics is a biopharmaceutical company that develops novel therapies for patients with rare copper-mediated disorders. Wilson Therapeutics’ product, WTX101, is in Phase 3 development as a treatment for Wilson disease, a rare genetic disorder with devastating hepatic and neurological consequences for patients. WTX101 is a first-in-class oral copper-binding agent with a unique mechanism of action and ability to access and bind copper from serum and promote its removal from the liver.
WTX101 has received Fast Track designation in the US and Orphan Drug Designation for the treatment of Wilson disease in the U.S. and EU. The transaction is expected to close in the second quarter of 2018
“Wilson disease is a rare disorder that can lead to severe liver disease, including cirrhosis and acute liver failure, as well as debilitating neurological morbidities such as impaired movement, gait, speech, swallowing, and psychiatric disorders. WTX101 is an innovative product that addresses the underlying cause of the disease and has the potential to define a new standard of care in treating Wilson disease, an area that has not had a new treatment in over two decades,” said Ludwig Hantson, Chief Executive Officer of Alexion. “The acquisition of Wilson Therapeutics is a strong strategic fit for Alexion given the overlap with our current clinical and commercial focus on metabolic and neurologic disorders, and is an important first step in rebuilding our clinical pipeline.”
“Alexion is a global leader in rare diseases with a proven record of developing and commercializing therapies for patients with rare diseases, making them a great partner to make WTX101 available to Wilson disease patients worldwide,” said Jonas Hansson, CEO of Wilson Therapeutics.
Wilson disease is a rare, chronic, genetic and potentially life-threatening liver disorder of impaired copper transport. Copper balance is normally maintained in the body by hepatic excretion of excessive copper in the bile. In patients with Wilson disease, a genetic mutation disables this biliary excretion pathway and excess copper accumulates over time in the liver cells. Wilson disease affects approximately one in every 30,000 people worldwide. The average age of diagnosis is 15-20 years, with the majority of patients presenting between the ages of 10 and 30.