Orphalan announces publication of results from the CHELATE

Orphalan announces publication of results from the CHELATE trial
in The Lancet Gastroenterology & Hepatology

Trial supported Orphalan’s successful application to the United States Food and Drug Administration (FDA) for approval of Cuvrior™ (trientine tetrahydrochloride) for Wilson’s disease

Paris, France 30 September, 2022 – Orphalan SA (“Orphalan” or “the Company”), an international orphan drug development and commercialization company, today announces the publication of the Company’s phase III CHELATE trial in The Lancet Gastroenterology & Hepatology¹, presenting the efficacy and tolerability of trientine tetrahydrochloride for the treatment of Wilson’s disease over a period of one year.

The CHELATE trial was the first prospective randomized trial comparing penicillamine with trientine tetrahydrochloride (TETA-4HCl), and supported Orphalan’s successful application to the United States Food and Drug Administration (FDA) for approval of Cuvrior™, an oral TETA-4HCl formulation for the treatment of adult patients with stable Wilson’s disease who are de-coppered and tolerant to penicillamine. It was the first treatment for Wilson’s disease to be approved by the FDA in over five decades.

During the trial, an innovative assay was developed to measure non-caeruloplasmin bound copper (NCC), the free and potentially toxic pool of copper in the blood. This assay measures NCC by separation and quantification of caeruloplasmin and its bound copper in serum with greater accuracy and precision than methods used in clinical practice today.

Using this NCC measurement, TETA-4HCl was determined to be non-inferior to penicillamine at the primary endpoint of the study (24 weeks), with the same observation at the end of the extension phase of the study (one year from randomization) in patients previously receiving maintenance penicillamine therapy.

Trial design and results

53 adult patients receiving maintenance penicillamine therapy for at least a…