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PANTHERx Rare announces release of treatment for rare stable Wilson Disease

pharmafile | April 20, 2023 | News story | Sales and Marketing  

US-based rare medicines pharmacy PANTHERx Rare has announced the launch of Cuvrior, a treatment for stable Wilson Disease in adults who are de-coppered and able to take penicillamine.

 

Wilson Disease is a rare genetic disorder which is caused by a mutation in the ATP7B gene. This results in the accumulation of copper throughout the body, which can lead to a wide range of symptoms such as tremours, anxiety, jaundice due to liver injury and Kayser-Fleischer rings (brown rings in the corneas of the eyes). Serious complications include: problems with the liver and kidneys; neurological issues that affect gait and speech; mental health problems like depression and psychosis; and blood disorders such as jaundice and anaemia.

 

Cuvrior works by chelating copper in the body to decrease copper absorption and increase the removal of the copper by urination. Clinical trials found Cuvrior to be well-tolerated and superior to penicillamine in reducing non-ceruloplasmin copper (NCC) levels.

 

Rob Snyder, president of PANTHERx Rare stated: “We are pleased to add Cuvrior to the list of PANTHERx medications. The PANTHERx team works hard to make a positive difference in the lives of our patients who are dealing with devastating and debilitating diseases and Cuvrior is yet another way we can make an impact.”

 

James Spargo


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