Phase 3 study will evaluate safety and efficacy of an investigational once-daily formulation of trientine tetrahydrochloride compared to D-penicillamine as first line treatment in patients with Wilson disease.

CHICAGO, Sept. 8, 2026 /PRNewswire/ — Orphalan Inc. today announced the launch of the TRADITiONAL Study, a global, multicenter Phase 3 clinical trial evaluating an investigational once-daily formulation of trientine tetrahydrochloride for the first line treatment of individuals with Wilson disease. The study will evaluate safety and efficacy of the investigational once-daily formulation of trientine tetrahydrochloride compared with D-penicillamine (DPA), an established copper-chelating treatment, as first line treatment in patients with Wilson disease, over 48 weeks following randomization.

“Successful management of Wilson disease requires lifelong therapy, and current treatment regimens can be complex and burdensome for many patients, posing significant challenges with adherence,” said Omar Kamlin, Chief Medical Officer, Orphalan. “The initiation of the global TRADITiONAL Study in the USA reflects Orphalan’s commitment in addressing the unmet needs of Wilson disease patients by investigating a therapeutic approach which may simplify the treatment burden.”

About the TRADITIONAL Study

TRADITiONAL is a global, multicenter, randomized, parallel-group, open-label Phase 3 study in symptomatic and asymptomatic Wilson disease patients aged 8 years and older who are either naïve to all Wilson disease therapies (treatment-naïve) or naïve to chelator therapy (symptomatic patients treated with zinc salts for ≤ 28 days may be eligible). Following approximately four weeks of screening, participants will receive 48 weeks of treatment. The study will evaluate efficacy, safety, tolerability, and patient-reported treatment satisfaction.

U.S. study sites include:

• University of Colorado Anschutz School of Medicine
• Yale University School of Medicine
• University of Michigan Medical Center

Additional sites are expected to join this year as global enrollment expands to China, Pakistan and Saudi Arabia.

More information is available on ClinicalTrials.gov (Identifier: NCT07465718).

About Wilson Disease

Wilson disease is a rare inherited disorder of copper metabolism caused by mutations in the ATP7B gene. The disease prevents the body from eliminating excess copper, leading to toxic accumulation in tissues and organs, primarily the liver and brain. Without lifelong treatment, Wilson disease can give rise to serious hepatic, neurologic, and psychiatric complications, and importantly, untreated Wilson disease can be fatal.

About Orphalan

Orphalan is a pharmaceutical company dedicated to developing and commercializing treatments for rare diseases, including Wilson disease. The company’s portfolio has recently expanded to include infantile epileptic spasms syndrome (IESS), a rare form of paediatric epilepsy, as well as neuro-oncology, focusing on conditions with significant unmet medical need. By combining scientific rigor with a strong patient-centered mindset, Orphalan works closely with healthcare professionals and patient communities to improve standards of care. Its model emphasizes deep medical engagement, long-term partnerships, and sustainable access to therapies, ensuring that people living with rare and often overlooked disorders receive tailored, specialized treatment options. Find out more at www.orphalan.com

GL-ORPH-CUPR-26036, September 2026

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SOURCE Orphalan