The goal of this clinical trial is to learn if a new trientine tetrahydrochloride (TETA 4HCl) formulation administered once a day compared to d-Penicillamine (DPA) as a first line treatment for people living with Wilson's disease (WD) is effective and safe. The study is enrolling children aged 8 years and older weighing at least 55 lb (25 kg) and adults with a recent diagnosis of WD. People recently diagnosed with WD, may be eligible for the study if they have either not started copper chelating treatment (such as DPA or trientine) or have been taking zinc salts for less than 28 days. Participants will be randomly allocated (like tossing a coin) to receive either DPA or TETA 4HCL for 48 weeks. During this time period participants will have up to 12 visits for health checks and assessments including blood and urine testing. In addition, at some visits participants may be asked to complete questionnaires on treatment satisfaction, and overall well-being.

Start Date01 Aug 2026

Sponsor / Collaborator

Orphalan SA

NCT07010575

/ CompletedPhase 2

Decentralized Study to Assess Patient Treatment Preference Comparing Their Current Standard-of-care Wilson's Disease (WD) Treatment to a New Trientine (TETA) 4HCl Formulation.

Decentralized study to assess patient reported treatment satisfaction comparing their current standard-of-care Wilson's Disease (WD) treatment with a new once-daily Trientine (TETA) 4HCl formulation.

Start Date15 Jul 2025

Sponsor / Collaborator

Orphalan SA

CTR20244688

/ CompletedNot Applicable

盐酸曲恩汀片在健康受试者中空腹生物等效性试验

[Translation] Fasting bioequivalence study of trientine hydrochloride tablets in healthy volunteers

主要目的：考察中国健康受试者在空腹状态下单次口服四川科瑞德制药股份有限公司生产的盐酸曲恩汀片与参比制剂体内药代动力学特征，评价两种制剂的生物等效性。次要目的：评价单次口服盐酸曲恩汀片受试制剂及参比制剂在中国健康受试者中的安全性。

[Translation]

Primary objective: To investigate the pharmacokinetic characteristics of the Trientine Hydrochloride Tablets produced by Sichuan Kred Pharmaceutical Co., Ltd. and the reference preparation in Chinese healthy subjects after a single oral administration under fasting conditions, and to evaluate the bioequivalence of the two preparations. Secondary objective: To evaluate the safety of the test preparation and the reference preparation of Trientine Hydrochloride Tablets in Chinese healthy subjects after a single oral administration.

Start Date28 Dec 2024

Sponsor / Collaborator

Sichuan Credit Pharmaceutical Co., Ltd.

100 Clinical Results associated with Trientine tetrahydrochloride

100 Translational Medicine associated with Trientine tetrahydrochloride

100 Patents (Medical) associated with Trientine tetrahydrochloride

258

Literatures (Medical) associated with Trientine tetrahydrochloride

01 Jun 2026·Clinical Case Reports

Pediatric Wilson Disease in Sudan: A Rare Case, Sudan Conflict and Diagnostic Challenges

Article

Author: Mehad Mortada BadrAlden Ahmed ; Sudad salahaldeen Hassan Mohammed ; Aprar Kamal Mohammed Ali Almamoon ; Ahmed Alshafei Elmahi Ahmed ; Fatima Bashir Omer Bashir ; Amjed Abdu Ali Mohammed ; Anaheed Alzubair Mohammed HabeebAllah ; Arafa Mubarak Abotalib Aref ; Tarig Mohamed Nourallah Altegani

ABSTRACT:

Wilson disease is rarely reported among African children. Wilson disease is due to a mutation in ATP7B on an autosomal recessive pattern, which causes defective copper excretion and copper accumulation in tissues such as liver and brain. To the best of our knowledge, this is the second reported case of Wilson disease from Sudan, and it highlights the challenges of diagnosis and management in low‐resource settings. Our report strengthens the need for awareness and accountability of rare diseases in public health systems, especially when access to diagnostics and special therapies is limited. A 13‐year‐old male of Sudanese descent with consanguineous parents presented with progressive generalized edema, jaundice, and ascites. On physical examination, Kayser‐Fleischer rings were observed. Laboratory tests revealed hypoalbuminemia, thrombocytopenia, and a family history of sibling deaths. Additionally, there was a difference in neuropsychiatric status. MRI of the brain demonstrated hyperintensities in the basal ganglia, known as the “giant panda sign.” A diagnosis of Wilson disease was made, with a Leipzig score of 7/10, based on low serum copper, hemolytic anemia, and the family history. Initial therapy with D‐penicillamine and then zinc was administered, but it failed to prevent neurological decline. This deterioration occurred between visits. Moreover, the only alternate therapy, trientine, in all its forms, was not available at the time. Wilson disease, thought rare, occurs in African children but remains underdiagnosed due to resource constraints. This case highlights diagnostic challenges, the critical role of clinical suspicion, and urgent need for accessible diagnostic and affordable therapies in low‐resource settings to improve outcome.

01 Apr 2026·INFLAMMOPHARMACOLOGY

Trientine ameliorates bleomycin-induced pulmonary fibrosis in rats through copper chelation and modulation of the CTR1/LOX/COL pathway

Article

Author: Radwan, Asmaa ; Elshal, Mahmoud ; Khalil, Ibrahim T ; Elsayed, Norhan M

Abstract:

Pulmonary fibrosis (PF) remains a devastating disease. Copper (Cu), an essential trace element, has been implicated in fibrotic processes in various organs, including the lungs. This study investigated the potential protective effects of trientine (TRI), a copper chelator, against bleomycin (BLM)-induced PF in rats and explored the underlying mechanisms. PF was induced by intratracheal administration of BLM (5 mg/kg). Rats were randomly allocated into seven groups: normal control, TRI alone, BLM control, and four BLM plus TRI prophylactic or therapeutic groups receiving low (55 mg/kg) or high (110 mg/kg) TRI doses orally. Results demonstrated that TRI dramatically reversed BLM-elevated serum levels of lactate dehydrogenase and alkaline phosphatase, markers of tissue damage. The protective effects of TRI were multifaceted: it exhibited potent antifibrotic activity by reducing collagen buildup and inhibiting TGF-β, CTR1, LOX, and COL1 expression; it acted as an antioxidant by increasing antioxidant status and significantly upregulating Nrf2; and it served as an anti-inflammatory agent by decreasing inflammatory cytokines (TNF-α and IL-6) and modulating the NF-κB pathway by increasing NF-κB expression while limiting its activation and nuclear translocation, thereby reducing inflammatory signaling. Histopathological and immunohistochemical analyses confirmed substantial attenuation of collagen deposition and structural lung damage. Overall, TRI demonstrated potent antifibrotic, antioxidant, and anti-inflammatory properties, effectively protecting lung tissue against BLM-induced injury. These findings highlight copper chelation as a promising therapeutic strategy for pulmonary fibrosis and warrant further investigation into TRI’s clinical potential.

01 Apr 2026·ADVANCES IN THERAPY

Patient Burden in the Treatment of Wilson Disease in the United States: An Analysis of Real-World Health Insurance Claims Data from the Komodo database

Article

Author: Strader, Carey ; Schultze, Michael ; Teynor, Megan ; Weiss, Karl Heinz ; Akumo, Divine ; Hedera, Peter ; Costantino, Halley

INTRODUCTION:

Wilson disease (WD) is a rare inherited disorder that causes copper accumulation and can be fatal if untreated. This study used real-world data from the US Komodo Health claims database to describe healthcare resource utilization (HCRU) and evaluate direct economic costs among patients with WD.

METHODS:

This retrospective observational study identified patients with WD using ICD-9/10 codes (excluding Menkes disease) between 2016 and 2019, with data spanning 2012-2020. Sociodemographic characteristics, HCRU, and costs were analyzed using SPSS v23, SAS v9.4, and R v3.6.0. The study was approved by Pearl Pathways IRB (#20-KANT-224).

RESULTS:

A total of 2115 patients with prevalent WD, including 360 ever-treated (with reimbursable WD prescriptions), were identified. During the 2-year follow-up, about 25% were hospitalized, with a mean stay of 9 days, and most visited the ER three times annually. Hepatic patients with WD were more likely to undergo liver biopsy or transplant but had fewer home health visits and less use of assistive mobility devices. Annual liver transplant costs averaged $9094.72 ± 8110.23 per prevalent WD patient and $10,147.98 ± 7030.83 per ever-treated patient. Mean annual costs per prevalent versus ever-treated patients with WD were inpatient ($716.52 ± 2675.06 vs. $252.75 ± 333.39), pharmacy ($270.35 ± 1348.79 vs. $1284.51 ± 2994.85), and outpatient ($73.93 ± 156.89 vs. $60.82 ± 62.17), respectively. Pharmacy costs for adherent patients averaged $157,505.28 for any medication, $252,617.11 for D-penicillamine, $189,328.52 for trientine, and $1574.91 for zinc. Among ever-treated patients with WD, respective costs were lower at $85,117.60, $123,190.73, $100,017.20, and $820.35.

CONCLUSION:

Estimated annual HCRU and treatment costs for patients with WD were lower than previously reported. These findings provide updated real-world insights into the economic burden of WD and highlight the cost implications of medication adherence in managing this rare disorder in the USA.

News (Medical) associated with Trientine tetrahydrochloride

08 Sep 2026

·www.prnewswire.com

Orphalan Initiates Global Phase 3 TRADITiONAL Study of Investigational Once-Daily Trientine Therapy in Wilson Disease

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 GL-ORPH-CUPR-26036, September 2026 SOURCE Orphalan 21% more press release views with Request a Demo

Phase 3

12 Dec 2025

·www.prnewswire.com

Orphalan announces acquisition of Orphelia Pharma

PARIS, Dec. 12, 2025 /PRNewswire/ -- Orphalan, an international pharmaceutical company specializing in the development and commercialization of orphan drugs, has acquired Orphelia Pharma, a company committed to treating rare and severe pediatric diseases, notably in neurology and oncology. This strategic transaction strengthens Orphalan's position as a European leader with a global commercial footprint in rare diseases, expanding its therapeutic portfolio. Key highlights of the acquisition include: Expansion of our capabilities in rare neurological and oncological pediatric diseases Greater reach and improved access to medicines for children and adults with rare conditions A unique platform combining scientific excellence, agility, and global commercialization By bringing together complementary expertise, the companies are now positioned to accelerate innovation and improve access to transformative treatments for patients around the world. As they enter this new stage, their teams remain fully committed to ensuring continuity of care and supporting their partners and patients. Dr. Naseem Amin, Chief Executive Officer of Orphalan said: "The acquisition of Orphelia Pharma reinforces our mission: to provide innovative and accessible treatments to patients living with rare diseases, wherever they are. "Together, we are building a unique platform that brings together scientific excellence, agility, international development and commercial capabilities to advance care for patients of all ages suffering from orphan diseases." Dr. Hugues Bienaymé, Founder and Deputy Chief Executive Officer of Orphelia Pharma, added: "At Orphelia Pharma, we have dedicated ourselves to developing safe and effective pediatric medicines that address major unmet medical needs in rare diseases. "Joining Orphalan will allow us to increase the impact of our treatments and make them available more rapidly to the children who need them most." Gilles Alberici, Chief Executive Officer of Orphelia Pharma, commented: "Bringing together our two complementary companies marks an important milestone, both for our organizations and for the broader community of patients living with rare diseases." About Orphalan Founded in 2011, Orphalan SA is an international pharmaceutical company dedicated to the development and commercialization of orphan drugs. Originating from a collaboration with AGEPS and Hôpital Lariboisière, Orphalan developed Cuprior®, a treatment for Wilson's disease, now authorized in 38 countries and marketed in 28. Orphalan's ambition is to reduce diagnostic delays and make innovative orphan drugs accessible to as many patients as possible through a patient-centered approach and close collaboration with healthcare professionals. Orphalan's shareholders include Bpifrance, Advent, and the management team. About Orphelia Pharma Orphelia Pharma is a pharmaceutical company based in Paris and Lyon, specializing in the development and commercialization of medicines for rare and severe pediatric diseases. With one authorized medicine in the European Union ( Kigabeq®) and a second under regulatory review ( Kizfizo®), Orphelia Pharma has established regional distribution agreements across Europe and other regions and collaborates with academic and industrial partners to advance research in rare diseases. Further information: Bien Commun Advisory M. Benedict Brodrick +33.6.76.14.02.02 [email protected] SOURCE Orphalan 21% more press release views with Request a Demo

AcquisitionDrug Approval

20 Aug 2025

·pharma.economictimes.indiatimes.com

Govt mulls tweaks in PRIP to boost rare disease therapies: Pharma Secy

New Delhi: In a bid to address the steep cost of imported rare disease therapies , the government is considering modifications to its Promotion of Research and Innovation in Pharma MedTech Sector ( PRIP ) scheme to support domestic development, said Amit Agrawal, Secretary, Department of Pharmaceuticals. Delivering the special guest address at the FICCI Rare Disease Conference 2025, Agrawal said, “Rare diseases in India are as significant as any other public health issue. While we are known as the pharmacy of the world, the need of the hour is to invest in research and development." “Under this vision we launched PRIP and we are at very final stages of making some tweaks to make it better and one of the key focus is the inclusion of rare diseases and orphan drugs,” he said adding that the government is considering rare diseases as a strategic focus with a higher policy support under the scheme.” Launched in 2023 with an allocation of Rs 5,000 crore, PRIP is a Research-Linked Incentive (RLI) initiative aimed at driving innovation in pharmaceuticals and MedTech. Of the scheme’s total outlay Rs 700 crore is earmarked under component ‘A’ for establishing 7 CoEs at NIPER and the rest Rs 4,250 crore is under component ‘B’ for promoting drug development in areas like New Chemical Entities, Complex generics including biosimilars, medical devices, stem cell therapy, orphan drugs, Anti-microbial resistance etc.” Agrawal also highlighted progress under the Production-Linked Incentive (PLI) scheme, noting that eight rare disease drugs—including Trientine (Wilson’s disease), Nitisinone (Type 1 Tyrosinemia), Eliglustat and Miglustat (Gaucher’s), Hydroxyurea (sickle cell anemia), Sapropterin (Phenylketonuria), Sodium Phenylbutyrate, and Carglumic Acid (Hyperammonemia) are now produced domestically. Stating that PLI scheme has brought down treatment costs significantly, he noted, “the annual cost of therapy for Gaucher’s disease has dropped from around Rs 3.6 crore to Rs 3 to 6 lakhs." The production of these drugs began in 2023 following the addition of pharmaceuticals in the PLI scheme with a financial outlay of ₹15,000 crore for the production period of fiscal year 2022-2023 to 2028-29. However, patient groups argue that the therapies currently covered are largely off-patent small molecules, whereas the the greater hurdle for patients is posed by advanced therapies such as large molecules, biologics, and gene therapies , that are still under patent protection. For instance, a disease-modifying therapy Risdiplam marketed by Swiss drugmaker Roche under the brand name Evrysdi for spinal muscular atrophy (SMA)–a neuromuscular disease–costs Rs 60–80 lakh annually, while Novartis’ one-time gene therapy Zolgensma (onasemnogene abeparvovec) entails Rs 14–16 crore. India’s National Policy for Rare Diseases (NPRD), introduced in 2021, has established 13 CoEs—including the latest at AIIMS Patna—to provide treatment and counselling for 63 listed rare diseases. According to the Ministry of Health and Family Welfare, as of August 2024, over 1,100 patients have benefited. Yet advocacy groups stress that the Rs 50 lakh financial support cap is inadequate for advanced therapies , and access to centers remains limited. By Abhijeet Singh ,

Gene Therapy

100 Deals associated with Trientine tetrahydrochloride

External Link

KEGG	Wiki	ATC	Drug Bank
D11649	-	A16AX12	-

R&D Status

10 top approved records.

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Indication	Country/Location	Organization	Date
Hepatolenticular Degeneration	European Union	Orphalan SA	05 Sep 2017
Hepatolenticular Degeneration	Iceland	Orphalan SA	05 Sep 2017
Hepatolenticular Degeneration	Liechtenstein	Orphalan SA	05 Sep 2017
Hepatolenticular Degeneration	Norway	Orphalan SA	05 Sep 2017

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT06128954 (TRIUMPH-3, EASL2026)	Phase 1	Hepatolenticular Degeneration	26	Trientine tetrahydrochloride 300 mg	wnecrriywy(fdkdladnac) = comparable systemic TETA AUC0-24 values observed for QD and BID TETA 4HCl respectively. bpwdsfcyas (ttbzcciyox ) View more	Positive	27 May 2026
				Trientine tetrahydrochloride 150 mg
NCT06128954 (TRIUMPH-3, CTgov)	Phase 1	Hepatolenticular Degeneration	26	900mg TETA 4HCl Once Daily Formulation (Once Daily Dose Formulation (Treatment A))	blrhzrutin(ptelomrvqb) = ulpxoqkvfr panyklhaet (dhkfgiiydw, zlqajhcpat - ppsgrsjioe) View more	-	15 Aug 2025
				900mg TETA 4HCl Cuprior® (Cuprior® Comparator (Treatment B))	blrhzrutin(ptelomrvqb) = pppmfaxtdk panyklhaet (dhkfgiiydw, mfifeqwvov - ctfncmokzq) View more
NCT03539952 (CHELATE, EASL2022)	Phase 3	Maintenance 24-h urinary copper excretion \| alanine aminotransferase	76	D-penicillamine (DPA)	kdqhoroukg(bnbdbxquwy) = intwcrcnoo wewcbyjyqa (iuzpqfynym )	Positive	24 Jun 2022
				trientine tetrahydrochloride	kdqhoroukg(bnbdbxquwy) = yifcgkhmyx wewcbyjyqa (iuzpqfynym )
NCT03539952 (EASL2021)	Phase 3	Hepatolenticular Degeneration Maintenance non-ceruloplasmin copper;(NCC)	53	D-penicillamine	gdcngwimvx(zxaaoqftrw) = wiydifuebu dyllljjpmb (pmfjvnobad )	Positive	23 Jun 2021
				TETA4	gdcngwimvx(zxaaoqftrw) = sqvbwmdtzf dyllljjpmb (pmfjvnobad )

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