Orphalan announces US launch of drug to treat Wilson’s disease
Orphalan SA, an international orphan drug development and commercialization company, has announced the commercial launch of Cuvrior in the US.
Cuvrior is a new trientine tetrahydrochloride (TETA-4HCl). It is now available for the treatment of adult patients with stable Wilson’s disease who are de-copper and tolerant to D-penicillamine.
Cuvrior was approved by the United States Food and Drug Administration (FDA) and granted Orphan Drug Exclusivity (ODE) in April 2022 for this group of patients.
Compared to standard of care, Cuvrior facilitates precise dosing by providing small, scored tablets in blister packs. Additionally, room temperature stability eliminates the need for refrigeration, making it a more practical solution for patients with busy lifestyles.
Orphalan drugs are available in Europe
Wilson’s disease is a rare inherited disorder of copper transport that primarily affects the liver and brain. Since 2019, Orphalan has been commercializing its trientine tetrahydrochloride product in Europe under the Cuprior name and around 1,000 Wilson’s disease patients have received the treatment.
Cuvrior was approved by the FDA based on data from the company’s phase III CHELATE trial, the first prospective randomized trial to compare penicillamine with TETA-4HCl. During the experiment, an assay was developed to measure non-caeruloplasmin bound copper (NCC), a collection of free and potentially toxic copper in the blood. Using this NCC measurement in patients previously receiving maintenance penicillamine therapy, TETA-4HCl was determined to be not inferior to penicillamine at the study’s primary end point (24 weeks), with similar observations at the end of the study extension phase. (one year from randomization).
Dmitry Paramonov, president of Orphalan AS, said: “We are excited to introduce Cuvrior in the US, a safe and innovative treatment option for patients and caregivers treating Wilson’s disease. Our distribution partners have Cuvrior in stock and ready to go immediately. At Orphalan, our focus is on providing targeted solutions for rare diseases, as demonstrated by the successful launch of Cuprior in Europe. We are eager to expand the availability of Cuvrior to as many patients as possible, underscoring our dedication to making a meaningful impact on the lives of rare disease patients around the world.”
About Wilson’s disease
Wilson’s disease is a rare genetic disease characterized by the gradual accumulation of copper from food over time, possibly to life-threatening levels. Affected individuals are unable to effectively excrete copper naturally through the digestive tract. Common symptoms associated with Wilson’s disease include progressive liver dysfunction, neurological disorders such as severe tremors, and decreased mental health. About 1 in 15 patients eventually requires a liver transplant. Wilson’s disease affects nearly 1 in every 30,000 people worldwide.
Several companies treat this rare disease. Wilson Therapeutics, which was acquired by Alexion Pharmaceuticals in an $855 million deal in 2018, reported positive results from the drug WTX101 in 2016.