Triethylenetetramine

Orphalan announces China NMPA’s approval of its trientine tetrahydrochloride product for the treatment of Wilson disease

Retrieved on: 
Thursday, January 18, 2024
Ageing, Patient, Long-term care, Disease, Wilson's disease, Liver, Triethylenetetramine, HCI, NMPA, DPA, Marketing, Brain, National Medical Products Administration, Diagnosis, Pharmaceutical industry

It offers a new treatment option for Wilson disease in children aged five years and older and adults intolerant to penicillamine therapy.

Key Points: 
  • It offers a new treatment option for Wilson disease in children aged five years and older and adults intolerant to penicillamine therapy.
  • China is home to an estimated 80,000 individuals with Wilson disease, of whom approximately half have their diagnosis confirmed, and 25,000 are receiving regular treatment.
  • Dr Naseem Amin, Chief Executive Officer at Orphalan, said: “We are pleased with the marketing authorisation approval of our trientine tetrahydrochloride product in China, which offers a well-tolerated and effective treatment option for patients with Wilson disease.
  • It is a significant step for those of us living with Wilson disease, representing a much-needed alternative treatment option.

PANTHERx® Rare Announces Release of CUVRIOR™ (trientine tetrahydrochloride) to Treat Wilson Disease

Retrieved on: 
Thursday, April 20, 2023
Triethylenetetramine, NCC, Tremor, Depression, Psychosis, Anxiety, Mutation, Liver injury, Jaundice, Genetic disorder, Patient, ATP7B, Anemia, Penicillamine, Liver, Copper, Disease

PITTSBURGH, April 19, 2023 /PRNewswire/ -- PANTHERx® Rare, a leader in rare disease product patient access and support services in the United States, is pleased to announce the launch of CUVRIOR to treat stable Wilson Disease in adults who are de-coppered and able to take penicillamine.

Key Points: 
  • PITTSBURGH, April 19, 2023 /PRNewswire/ -- PANTHERx® Rare, a leader in rare disease product patient access and support services in the United States, is pleased to announce the launch of CUVRIOR to treat stable Wilson Disease in adults who are de-coppered and able to take penicillamine.
  • Wilson Disease is a rare genetic disorder caused by a mutation in the ATP7B gene, resulting in the accumulation of copper throughout the body.
  • CUVRIOR is an innovative oral formulation of the active ingredient trientine created by Orphalan to treat adult patients with stable Wilson Disease who have already been de-coppered.
  • In clinical trials, CUVRIOR was found to be well tolerated and non-inferior to penicillamine in reducing non-ceruloplasmin copper (NCC) levels in Wilson Disease patients.

Global Wilson's Disease Drugs Market to 2030: Players Include Kadmon Holdings, Merck & Co, Noble Pharma and Teva Pharmaceutical Industries - ResearchAndMarkets.com

Retrieved on: 
Friday, April 14, 2023
Biotechnology, Pharmaceutical, Health, Seizure, Triethylenetetramine, Male, Fatigue, Psychosis, Hepatitis, Partnership, Wilson's disease, Tremor, Zinc, Prevalence, Schizophrenia, Ataxia, Mutation, Dystonia, Zinc acetate, Brain, Data mining, Merck & Co., DSM-IV codes, Person, Wilson, Dietary supplement, Patient, Drug discovery, Liver, Diagnosis, Copper, Basal ganglia, News, Bipolar disorder, Disease, Penicillamine, Female, Pharmaceutical industry, Parkinsonism

It is very important for the accurate diagnosis of Wilson's disease or could lead to fatal consequences if not done appropriately.

Key Points: 
  • It is very important for the accurate diagnosis of Wilson's disease or could lead to fatal consequences if not done appropriately.
  • The early diagnosis of Wilson's disease is primarily based on the multiple indications such as hepatic, neuropsychiatric, ophthalmic and others.
  • Product portfolio gives an idea about the different dietary supplements enriched in zinc primarily used for the management of Wilson's disease.
  • The manufacturers that are active in the treatment of Wilson's disease are Kadmon Holdings, Inc., Merck & Co., Noble Pharma Co., Ltd., Teva Pharmaceutical Industries Limited, Tsumura & Co., Valeant Pharmaceuticals International, Inc., VHB Life Sciences, Inc. and Wilson Therapeutics AB.

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

Retrieved on: 
Friday, September 30, 2022
Penicillamine, Adult, Observation, Yale University, Brain, FDA, Disease, Center, Blood, Excellence, LinkedIn, Triethylenetetramine, Center of excellence, Wilson, Maintenance, Patient, Copper, Physician, III, Ceruloplasmin, Serum, Genetic disorder, SAE, Liver, NCC, Method, Degenerative disease, Research, Unified, Wilson's disease, Șaeș, Pharmaceutical industry, Plumbing, Medical imaging

It was the first treatment for Wilson's disease to be approved by the FDA in over five decades.

Key Points: 
  • 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.
  • The results of the CHELATE trial mark an important milestone for the Wilsons disease community, providing further evidence on the efficacy of trientine tetrahydrochloride for prescribing physicians and evidence-based options for patients.
  • Orphalan commercializes Cuprior, its trientine tetrahydrochloride product for the treatment of Wilsons disease in Europe, and will be launching Cuvrior, recently approved by the FDA, in the US.

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

Retrieved on: 
Friday, September 30, 2022
Penicillamine, Adult, Observation, Yale University, Brain, FDA, Disease, Center, Blood, Excellence, LinkedIn, Triethylenetetramine, Center of excellence, Wilson, Maintenance, Patient, Copper, Physician, III, Ceruloplasmin, Serum, Genetic disorder, SAE, Liver, NCC, Method, Degenerative disease, Research, Unified, Wilson's disease, Șaeș, Pharmaceutical industry, Plumbing, Medical imaging

It was the first treatment for Wilson's disease to be approved by the FDA in over five decades.

Key Points: 
  • 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.
  • The results of the CHELATE trial mark an important milestone for the Wilsons disease community, providing further evidence on the efficacy of trientine tetrahydrochloride for prescribing physicians and evidence-based options for patients.
  • Orphalan commercializes Cuprior, its trientine tetrahydrochloride product for the treatment of Wilsons disease in Europe, and will be launching Cuvrior, recently approved by the FDA, in the US.

Orphalan announces enrolment of the first five patients in the International Wilson's Disease Patient Registry (“iWD Registry”)

Retrieved on: 
Tuesday, July 26, 2022
Brain, Central Institute of Psychiatry, Phenotype, Physician, Triethylenetetramine, Institute, Genetic disorder, Natural history, Liver, Disease, FDA, MD, Patient, Neurology, LinkedIn, Psychiatry, Pharmaceutical industry, Doctor of Philosophy, Q4, MBBS

Paris, France [26 July], 2022 -- Orphalan SA (“Orphalan” or “the Company”), an international orphan drug development and commercialization company, today announces the enrolment of the first five patients into the International Wilson's Disease Patient Registry (“iWD Registry”), the first global registry of patients with Wilson’s Disease.

Key Points: 
  • Paris, France [26 July], 2022 -- Orphalan SA (Orphalan or the Company), an international orphan drug development and commercialization company, today announces the enrolment of the first five patients into the International Wilson's Disease Patient Registry (iWD Registry), the first global registry of patients with Wilsons Disease.
  • By including prospective data from patients worldwide, this registry will help physicians collaborate to find better standards for the treatment of Wilsons Disease.
  • Professor Pramod Mistry, MBBS, PhD, MA, MD, Director of Yale Lysosomal Disease Centre, added: An international registry of Wilsons Disease patients is long overdue, and I congratulate the team at Orphalan for establishing it.
  • The registry will also harness patient participation because there will always be questions from patients that individual centres cannot answer.

Orphalan announces enrolment of the first five patients in the International Wilson's Disease Patient Registry (“iWD Registry”)

Retrieved on: 
Tuesday, July 26, 2022
Brain, Central Institute of Psychiatry, Phenotype, Physician, Triethylenetetramine, Institute, Genetic disorder, Natural history, Liver, Disease, FDA, MD, Patient, Neurology, LinkedIn, Psychiatry, Pharmaceutical industry, Doctor of Philosophy, Q4, MBBS

Paris, France [26 July], 2022 -- Orphalan SA (“Orphalan” or “the Company”), an international orphan drug development and commercialization company, today announces the enrolment of the first five patients into the International Wilson's Disease Patient Registry (“iWD Registry”), the first global registry of patients with Wilson’s Disease.

Key Points: 
  • Paris, France [26 July], 2022 -- Orphalan SA (Orphalan or the Company), an international orphan drug development and commercialization company, today announces the enrolment of the first five patients into the International Wilson's Disease Patient Registry (iWD Registry), the first global registry of patients with Wilsons Disease.
  • By including prospective data from patients worldwide, this registry will help physicians collaborate to find better standards for the treatment of Wilsons Disease.
  • Professor Pramod Mistry, MBBS, PhD, MA, MD, Director of Yale Lysosomal Disease Centre, added: An international registry of Wilsons Disease patients is long overdue, and I congratulate the team at Orphalan for establishing it.
  • The registry will also harness patient participation because there will always be questions from patients that individual centres cannot answer.

ALXN1840 Shows Rapid and Sustained Improvement in Copper Mobilization From Tissues, Potentially Closing Treatment Gaps for Wilson Disease Community

Retrieved on: 
Thursday, June 23, 2022
Biotechnology, Pharmaceutical, Health, People, Nephrology, Biopharmaceutical, Degenerative disease, Union, Liver disease, Chelation therapy, Focus, Prior, Disease, Penicillamine, ANCOVA, Wilson's disease, Copper, Neurology, Hematology, European Association for the Study of the Liver, Clinician Administered PTSD Scale, WTX101, Data, Review, Immunology, Tissue, Death, ICL, Lists of diseases, Brain, Metabolism, Alexion Pharmaceuticals, Phase, Standard of care, LSM, American Association, SOC, Orphan, Patient, Diagnosis, AES, Blood, NCT, Association, EASL, AstraZeneca, Psychiatry, Safety, Liver, Alexion, Cardiology, Research, Ophthalmology, Dhaka, Triethylenetetramine, Acquisition, AE, Oral medicine, Medical device, Pharmaceutical industry

Cohort 1= Prior WD Treatment >28 days; Cohort 2= Treatment Nave or Prior WD Treatment 28 days.

Key Points: 
  • Cohort 1= Prior WD Treatment >28 days; Cohort 2= Treatment Nave or Prior WD Treatment 28 days.
  • Most adverse events (AEs) were not considered serious (ALXN1840, 85.4%; SoC, 75.7%) and/or were not considered related to trial treatment (ALXN1840, 77.4%; SoC, 75.7%).
  • The most common AE associated with ALXN1840 was a reversible increase in alanine aminotransferase levels (ALXN1840, 14.6%; SoC, 2.9%).
  • ALXN1840 has been granted Orphan Drug Designation in the United States and orphan designation in the European Union for Wilson disease.

Orphalan announces FDA approval of Cuvrior™ for the treatment of adult patients with stable Wilson’s disease who are de-coppered and tolerant to penicillamine

Retrieved on: 
Monday, May 2, 2022
Hope, Degenerative disease, Brain, Orphan, Center of excellence, NDA, Excellence, Penicillamine, Patient, III, FDA, Bureau of Oceans and International Environmental and Scientific Affairs, New Drug Application, MD, Wilson Disease Association, Adult, Center, LinkedIn, Triethylenetetramine, Liver, NCC, Safety, Yale University, Pharmaceutical industry

Cuvrior is approved for the treatment of adult patients with stable Wilsons disease who are de-coppered and tolerant to penicillamine.

Key Points: 
  • Cuvrior is approved for the treatment of adult patients with stable Wilsons disease who are de-coppered and tolerant to penicillamine.
  • Penicillamine is currently approved as a first-line treatment of Wilsons disease in the US with about one third of patients developing intolerance1.
  • Dr. Naseem Amin, Chief Executive Officer at Orphalan, commented: We are delighted with the approval of our product, Cuvrior, which provides a well-tolerated and effective option for Wilsons disease patients.
  • It has been approved under the 505(b)(2) pathway for the treatment of adult stable Wilsons disease patients who are successfully de-coppered and tolerant to penicillamine.

Orphalan announces FDA approval of Cuvrior™ for the treatment of adult patients with stable Wilson’s disease who are de-coppered and tolerant to penicillamine

Retrieved on: 
Monday, May 2, 2022
NCC, Adult, LinkedIn, Penicillamine, Yale University, III, Patient, FDA, Orphan, Degenerative disease, Triethylenetetramine, New Drug Application, MD, Bureau of Oceans and International Environmental and Scientific Affairs, Center, Excellence, Brain, NDA, Liver, Wilson Disease Association, Safety, Hope, Center of excellence, Pharmaceutical industry

Cuvrior is approved for the treatment of adult patients with stable Wilsons disease who are de-coppered and tolerant to penicillamine.

Key Points: 
  • Cuvrior is approved for the treatment of adult patients with stable Wilsons disease who are de-coppered and tolerant to penicillamine.
  • Penicillamine is currently approved as a first-line treatment of Wilsons disease in the US with about one third of patients developing intolerance1.
  • Dr. Naseem Amin, Chief Executive Officer at Orphalan, commented: We are delighted with the approval of our product, Cuvrior, which provides a well-tolerated and effective option for Wilsons disease patients.
  • It has been approved under the 505(b)(2) pathway for the treatment of adult stable Wilsons disease patients who are successfully de-coppered and tolerant to penicillamine.