trientine / Generic mfg. - LARVOL DELTA

Recent advances in the study of cuproptosis in gliomas. (PubMed, Int Immunopharmacol) - "Copper chelators, such as trientine and disulfiram, can regulate intracellular copper levels and induce glioma cell death, thereby enhancing the efficacy of chemotherapy. However, the specific mechanisms of cuproptosis and its clinical application in glioma treatment require further exploration. This field of study promises to offer novel insights and directions for developing more effective glioma therapies."

Journal • Review • Brain Cancer • Glioma • Oncology • Solid Tumor • FDX1 • LIAS

Patient Preference Study: Standard of Care Versus Once-daily Trientine Tetrahydrochloride (clinicaltrials.gov) - P2 | N=10 | Active, not recruiting | Sponsor: Orphalan | Recruiting ➔ Active, not recruiting

Enrollment closed • Genetic Disorders • Metabolic Disorders • Movement Disorders

Management of Wilson disease across Europe: an international physician-oriented survey by the ERN-RARE Liver group. (PubMed, Orphanet J Rare Dis) - "Overall, we found uniformity in the management of WD across European WD centers. Nevertheless, variations in key areas were identified, reflecting a lack of robust evidence, thus providing a guide for future research."

Journal • Genetic Disorders • Hepatology • Metabolic Disorders • Movement Disorders • Rare Diseases

Safety of penicillamine and trientine in the treatment of Wilson's disease: An analysis of the FDA Adverse Event Reporting System (FAERS) database. (PubMed, PLoS One) - "This study reveals the characteristics of AEs and potential associated risks in the clinical application of penicillamine and trientine, emphasizing individualized medication and vigilant monitoring strategies to provide guidance for safe medication use."

Adverse events • Journal • Alzheimer's Disease • CNS Disorders • Gastroenterology • Gastrointestinal Disorder • Genetic Disorders • Immunology • Metabolic Disorders • Movement Disorders

Rare liver diseases - Etiology, diagnosis and management: A review. (PubMed, Biomol Biomed) - "Lifelong copper chelation is recommended for Wilson disease, with trientine preferred for neurologic phenotypes. Supportive care in alpha-1 antitrypsin deficiency (A1ATD) is complemented by the investigation of molecular chaperones. Additionally, gene-directed therapies, gene editing, RNA-based approaches, and cell therapies show early promise but raise concerns regarding durability, safety, and ethical considerations, particularly for pediatric patients. In conclusion, implementing precision medicine frameworks that rely on standardized diagnostics, multicenter registries, and equitable access is crucial for facilitating earlier detection and translating mechanism-targeted therapies into sustainable, globally accessible benefits."

Journal • Review • Alpha-1 Antitrypsin Deficiency • Cholestasis • Genetic Disorders • Hepatology • Immunology • Metabolic Disorders • Movement Disorders • Pediatrics • Pulmonary Disease • Respiratory Diseases

Adherence, satisfaction, and quality of life in Wilson disease patients after switching to trientine tetrahydrochloride: observational data from a dual cohort study. (PubMed, Front Pharmacol) - "All quality-of-life (QoL) domains improved between T0 and T1. Adults with WD on maintenance therapy following a switch in therapy to TETA 4HCl some improvements in adherence, satisfaction and QoL were observed."

Clinical • HEOR • Journal • Observational data • Genetic Disorders • Metabolic Disorders • Movement Disorders

Late Onset Wilson Disease Presenting With Elevated Transaminases and Abnormal Ultrasound (ACG 2025) - "Repeat labs 6 weeks after initiating trientine show improvement of both AST and ALT, 45 U/L and 94 U/L respectively...In our patient, obesity initially directed the evaluation toward fatty liver disease...H&E StainFigure: Figure 2. Trichrome Stain"

Fibrosis • Genetic Disorders • Hepatology • Immunology • Inflammation • Liver Failure • Metabolic Disorders • Movement Disorders • Obesity • Ophthalmology • Psychiatry • ATP7B

Reprogramming the Tumor Microenvironment with Copper Chelators to Enhance Immunotherapy in Mesothelioma (IMIG 2025) - "Given their established safety profile and clinical use in childhood disorders, Cu chelators such as Trientine (TETA) represent a promising therapy to combine with immune checkpoint blockade (ICB) for mesothelioma. Ongoing phase I/II trials are evaluating Cu chelators in combination with anti-PD-1 (Pembrolizumab) and chemotherapy in breast cancer, or in combination with anti-GD2 (Dinutuximab)in neuroblastoma. These findings provide a strong rationale to assess the safety and feasibility of combining TETA with dual ICB (Ipilimumab/Nivolumab) in patients with mesothelioma."

Biomarker • IO biomarker • Tumor microenvironment • Breast Cancer • Gastric Cancer • Lung Cancer • Mesothelioma • Neuroblastoma • Oncology • Solid Tumor • CD8 • PD-L1

Wilson's Disease Initially Misdiagnosed as Metabolic Dysfunction-Associated Steatohepatitis in a Young Male (ACG 2025) - "He was started on Trientine HCl (500 mg AM & 750 mg PM). Our case underscores the complexity of diagnosis of Wilson's disease when hepatic steatosis is the only prominent finding...A broader differential, including Wilson's disease, should be considered in younger adults with elevated liver enzymes, even when imaging suggests MASH. Early diagnosis through comprehensive Cu studies allows for timely chelation therapy, helping to prevent irreversible liver or neurologic damage.Figure: Table 1: Laboratory ReportFigure: Table 2: Differential Diagnosis of Steatosis"

Fibrosis • Genetic Disorders • Hepatology • Immunology • Metabolic Disorders • Metabolic Dysfunction-Associated Steatohepatitis • Movement Disorders • ATP7B • CP

Behavioral and Quality of Life Challenges in Pediatric Wilson Disease: Insights From a Multisite International Registry (AACAP 2025) - "Participants on chelating agents (D-penicillamine or trientine) reported lower physical functioning (77.7 + 20.93, p = .004) and physical health (77.59 + 21.28, p = .005) than those on zinc (94.06 ± 7.35), possibly due to selection bias of using zinc for less ill patients. Children with WD experience internalizing symptoms (anxiety, depression, and somatic complaints) that impact their emotional well-being, social interactions, and academic performance. Children with WD experience internalizing symptoms (anxiety, depression, and somatic complaints) that impact their emotional well-being, social interactions, and academic performance. Females exhibiting more emotional distress may benefit from gender-sensitive interventions. The relationship between internalizing symptoms and academic struggles underscores the need to address mental health challenges early."

Clinical • HEOR • CNS Disorders • Depression • Genetic Disorders • Hepatology • Metabolic Disorders • Mood Disorders • Movement Disorders • Pediatrics • Psychiatry

Recommendations from the European Association for the Study of the Liver and the European Reference Network for Rare Liver Diseases Clinical Practice Guidelines for hepatolenticular degeneration (PubMed, Zhonghua Gan Zang Bing Za Zhi) - "Pharmacotherapy mainly includes chelating agents (such as penicillamine and trientine) and zinc salts...The diagnosis and treatment of acute liver failure with WD is extremely challenging, as the diagnosis is difficult and medical treatment cannot save life. The role of liver transplantation has been clearly recognized in the treatment of acute liver failure with WD, and it may also be considered in cases with neurological involvement."

Clinical guideline • Journal • Genetic Disorders • Hepatology • Liver Failure • Metabolic Disorders • Movement Disorders • Transplantation

MEDICATION ADHERENCE IN WILSON DISEASE PATIENTS TREATED WITH TRIENTINE TETRAHYDROCHLORIDE SUPPLIED BY A SINGLE SOURCE SPECIALTY PHARMACY (AASLD 2025) - "Most frequently reported were trientine hydrochloride capsules, zinc salts and penicillamine in that order. In this real-world analysis of patients with WD receiving TETA-4HCl through a single-source specialty pharmacy, adherence was consistently high with minimal interruption in therapy as described with a gap of 2.7 days per shipment and a low overall discontinuation rate of 17%. The extent to which single-source pharmacy distribution mitigates barriers to access, reduces treatment burden, and facilitates adherence warrants further investigation."

Adherence • Clinical • Genetic Disorders • Hepatology • Metabolic Disorders • Movement Disorders • Rare Diseases

TEMPEST: The Efficacy and Mechanism of Trientine in Patients With Hypertrophic Cardiomyopathy (clinicaltrials.gov) - P2 | N=154 | Completed | Sponsor: Manchester University NHS Foundation Trust | Active, not recruiting ➔ Completed

Trial completion • Cardiomyopathy • Cardiovascular • Hypertrophic Cardiomyopathy

TDMQ20 as A Drug Candidate for Wilson's Disease: Comparison with D-Penicillamine, Trientine, and Tetrathiomolybdate In Vitro and In Mice. (PubMed, Pharmaceutics) - "Conversely, DPA, TETA, and bcTTM give rise to various complexes with copper ions, often with oligomeric or cluster structures that can be retained in blood circulation or sequestered by proteins. Taking into consideration all the advantages of TDMQ20 compared to other ligands, including its lack of toxicity during long-term administration in mice, the drug candidate TDMQ20 appears to be a first-class challenger to the currently used treatments, i.e., DPA, TETA, and bcTTM."

Journal • Preclinical • Genetic Disorders • Metabolic Disorders • Movement Disorders

Wilson's Disease in Oman: A National Cohort Study of Clinical Spectrum, Diagnostic Delay, and Long-Term Outcomes. (PubMed, Clin Pract) - "Chelation therapy with trientine or penicillamine, often combined with zinc, was the mainstay of treatment...Early detection through cascade screening and sustained treatment adherence are critical for favorable outcomes. These findings support the need for national screening policies and structured long-term care models for WD in the region."

Journal • Genetic Disorders • Metabolic Disorders • Movement Disorders • Psychiatry

Patient Preference Study: Standard of Care Versus Once-daily Trientine Tetrahydrochloride (clinicaltrials.gov) - P2 | N=10 | Recruiting | Sponsor: Orphalan | Trial primary completion date: Aug 2025 ➔ Nov 2025

Trial primary completion date • Genetic Disorders • Metabolic Disorders • Movement Disorders

Patient Preference Study: Standard of Care Versus Once-daily Trientine Tetrahydrochloride (clinicaltrials.gov) - P2 | N=10 | Recruiting | Sponsor: Orphalan | Not yet recruiting ➔ Recruiting

Enrollment open • Genetic Disorders • Metabolic Disorders • Movement Disorders

Trientine-Induced Enteritis in Wilson Disease Treatment. (PubMed, J Hepatol) - No abstract available

Journal • Gastrointestinal Disorder • Genetic Disorders • Metabolic Disorders • Movement Disorders

The Economic Burden, Epidemiological Insights, and Treatment Patterns of Wilson's Disease: A Real-World Study in Italy. (PubMed, Drugs Real World Outcomes) - "The economic burden of WD in Italy varies with disease severity and treatment strategy, highlighting the need for optimized management practices to mitigate costs while enhancing patient care."

HEOR • Journal • Real-world evidence • Genetic Disorders • Metabolic Disorders • Movement Disorders

Pitfalls in the Diagnosis of Wilson Disease. (PubMed, Curr Neurol Neurosci Rep) - "Treatment is lifelong and includes chelating agents (penicillamine and trientine) and inhibitors of copper absorption (zinc salts). Liver transplant is an option for patients with end-stage liver disease. The key to successful therapy is early diagnosis."

Journal • Review • CNS Disorders • Genetic Disorders • Hepatology • Liver Failure • Mental Retardation • Metabolic Disorders • Movement Disorders • Psychiatry • Transplantation • ATP7B

Patient Preference Study: Standard of Care Versus Once-daily Trientine Tetrahydrochloride (clinicaltrials.gov) - P2 | N=10 | Not yet recruiting | Sponsor: Orphalan

New P2 trial • Genetic Disorders • Metabolic Disorders • Movement Disorders

Erratum: Effects of trientine and penicillamine on intestinal copper uptake: A mechanistic 64Cu PET/CT study in healthy humans. (PubMed, Hepatology) - No abstract available

Journal

ATP7B mRNA therapy for the treatment of Wilson's disease (WD) (ASGCT 2025) - "Current treatment options for WD, including copper-chelating agents like D-penicillamine and Trientine, have limited efficacy and are associated with numerous adverse effects...Collectively, our findings provide strong preclinical proof-of-concept for systemic mRNA-LNP as a potential disease-modifying therapy for patients with WD. Disease Focus of Abstract:Rare Diseases"

Gene Therapies • Genetic Disorders • Hepatology • Metabolic Disorders • Movement Disorders • Ophthalmology • Rare Diseases • ATP7B

Epidemiology, clinical presentation and management of Wilson's disease in Portugal – data from a national registry (EASL 2025) - "Around two-thirds of patients were initially treated with penicillamine, 18% with trientine and 16% with zinc... Our estimated prevalence of WD in the Portuguese population was similar to the numbers reported in the literature (1 : 30,000-50,000). The course of WD was characterized by multiple therapeutic regimens and therefore needs close monitoring for adhesion and side effects, since in fact one fifth of the patients finally needed LT, although under follow-up and treatment. This was the first nationwide epidemiologic study of WD in Portugal."

Clinical • Fibrosis • Genetic Disorders • Hepatology • Immunology • Inflammation • Liver Cirrhosis • Liver Failure • Metabolic Disorders • Movement Disorders

Management of Wilson disease across europe: an international physician-oriented survey by the ERN-RARE liver group (EASL 2025) - "In conclusion, this physician-oriented survey shows adherence to international WD guidelines among European centers. The survey also uncovers important differences amongst centers particularly related to the initial treatment of non-hepatological WD, availability of trientine and recommendations for low copper diet. The survey highlights numerous areas in WD care in which evidence is lacking."

Genetic Disorders • Hepatology • Metabolic Disorders • Movement Disorders