Trientine tetrahydrochloride (Cuprior, gmp-orphan SA) has been recommended for approval in Europe for the treatment of Wilson's disease in adults, adolescents, and children aged 5 years and older who are intolerant to d-penicillamine.
The positive opinion, from the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP), is for a hybrid medicine: a drug that is similar to an authorized medicine containing the same active substance but that differs in characteristics such as strength, indication, or pharmaceutical form. Hybrid applications rely in part on the results of preclinical tests and clinical trials for a reference product and in part on new data.
Wilson's disease is a rare genetic storage disease caused by a defect in a copper transporter gene, leading to copper accumulation in the liver, brain, eye, and peripheral nerves.
Trientine is a copper-chelating agent that removes copper from the body by forming a stable complex that is eliminated through urinary excretion. Trientine may also inhibit copper absorption from the intestinal tract.
Cuprior is a hybrid of trientine dihydrochloride, which has been on the market in Europe since 1985.
Cuprior works in the same way as the reference product but, unlike the reference product, the trientine salt in Cuprior (tetrahydrochloride) does not need to be stored in the refrigerator.
Cuprior has had orphan status since 2015. It will be available in 150-mg tablets.
"The benefits with Cuprior are its ability to decrease serum copper levels in patients with Wilson's disease," the EMA said in a statement.
Nausea is the most commonly reported adverse reaction with trientine. Serious iron deficiency anemia and severe colitis may occur during treatment, the agency notes.