Orphalan Expands Global Phase 3 TRADITiONAL Study in Wilson Disease to China Marking First Patient Randomised Worldwide

• Phase 3 trial evaluates the safety and efficacy of an investigational once-daily formulation of trientine tetrahydrochloride compared to D-penicillamine as first-line treatment in Wilson disease.
  
  
• First participant globally randomised in China at The Second Affiliated Hospital of Zhejiang University School of Medicine, following activation of three leading clinical centres with Beijing Friendship Hospital serving as coordinating lead site.

PARIS, Nov. 12, 2026 /PRNewswire/ -- Orphalan SA today announced the expansion of the TRADITiONAL Study into China, marking the formal randomisation of the first participant into the study globally. TRADITiONAL is an international, multicentre Phase 3 clinical trial evaluating an investigational once-daily formulation of trientine tetrahydrochloride for the first-line treatment of Wilson disease. The study evaluates the safety and efficacy of once-daily trientine tetrahydrochloride compared with D-penicillamine (DPA), an established copper-chelating therapy, over 48 weeks post randomisation.

The global study launched initially in the United States in August 2026 with a progressive activation of sites across the US and the King Faisal Specialist Hospital, Riyadh, Saudi Arabia. Additional sites expected to join this year as global enrolment expands to Pakistan.

This important milestone in China represents the first international study expansion in Wilson disease, with the study's first participant worldwide randomised at The Second Affiliated Hospital of Zhejiang University School of Medicine. Patient recruitment is now actively open across three leading Chinese hospital institutions, with Beijing Friendship Hospital serving as the coordinating lead site:

• Beijing Friendship Hospital, Capital Medical University (lead site)
• The Second Affiliated Hospital, Zhejiang University School of Medicine (site of the first randomised participant)
• Beijing You'an Hospital, Capital Medical University (activated clinical study site)

"The activation of centres in China and the randomisation of our first participant represent a significant milestone in our goal to develop innovative products addressing the current treatment burden of people living with Wilson disease. Our investigational once-daily formulation will be compared with D-penicillamine as a first-line therapy. Current treatment approaches can be complex for people living with Wilson disease. Our goal is to address the unmet patient needs by investigating options that may provide better support for the daily therapeutic management of Wilson disease while maintaining effective copper control." - Omar Kamlin, Chief Medical Officer, Orphalan

Notes to Editors

About the TRADITiONAL Study

TRADITiONAL is a global, multicentre, randomised, 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.

Global enrolment is active in the United States and China, with additional international study sites expected to join. 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, neurological, and psychiatric complications, and importantly, untreated Wilson disease can be fatal.

About Orphalan

Orphalan is a global pharmaceutical company dedicated to developing and commercialising 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 rigour with a strong patient-centred mindset, Orphalan works closely with healthcare professionals and patient communities to improve standards of care. Its model emphasises deep medical engagement, long-term partnerships, and sustainable access to therapies, ensuring that people living with rare and often overlooked disorders receive tailored, specialised treatment options. Find out more at www.orphalan.com

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SOURCE Orphalan