Orphalan

Orphalan S.A. is a privately held French biotechnology and orphan-drug company headquartered in Paris. Founded in 2011, the company focuses on the development, regulatory approval, and commercialization of treatments for patients with rare diseases. Its principal product is trientine tetrahydrochloride, a copper-chelating medicine used in the management of Wilson's disease, a rare inherited disorder in which impaired copper metabolism can cause copper accumulation in the liver, brain, and other organs. The company's trientine tetrahydrochloride product has been marketed under different names in different jurisdictions. It is known as Cuprior in the European Union and certain other markets, Cuvrior in the United States, and 科佩欧 (Ke Pei Ou) in China. The product's regulatory history has been central to Orphalan's development as a specialist rare-disease company. In the United States, the product received orphan-drug designation from the Food and Drug Administration in 2016 for Wilson's disease in patients who were not intolerant to penicillamine. In Europe, the European Medicines Agency granted marketing authorization in 2017 for adults, adolescents, and children aged five years and older who were intolerant to D-penicillamine therapy. Commercial European availability began in 2019. Orphalan subsequently expanded the product's regulatory and commercial reach. The U.S. Food and Drug Administration approved Cuvrior in April 2022 for adult patients with stable Wilson's disease who had been de-coppered and were tolerant to penicillamine. The approval was supported by the CHELATE clinical trial, and the product received U.S. orphan-drug exclusivity. U.S. commercial launch followed in April 2023. China’s National Medical Products Administration approved the medicine in December 2023 for Wilson's disease in adults, adolescents, and children aged five years and older who were intolerant to D-penicillamine; the Chinese commercial launch took place in March 2025. The medicine has been reported as available in more than 30 countries. Beyond medicine commercialization, Orphalan has supported disease-focused infrastructure. In 2021, it established the International Wilson Disease Registry, a centralized database sponsored and managed by the company that collects health information from consenting people with Wilson's disease in multiple countries. The registry is intended to support a better understanding of the disease and its treatment in routine clinical practice, although the available reference material does not specify its full research outputs or governance arrangements. Orphalan's positioning is that of a specialist orphan-drug developer and commercial operator rather than a diversified pharmaceutical conglomerate. The available material identifies trientine tetrahydrochloride as its principal product and Wilson's disease as its clearest therapeutic focus. It does not provide sufficient information to identify additional marketed products, a broader pipeline, ownership structure, financial performance, or a complete international management team. The company has nevertheless developed an international footprint through regulatory approvals and launches in Europe, the United States, and China, while retaining its French corporate base.

Orphalan S.A. was founded in France in 2011 as a company dedicated to orphan-drug development and commercialization. Its best-documented therapeutic program concerns Wilson's disease, a rare inherited disorder involving impaired copper handling. The disease can result in copper accumulation in tissues and requires long-term medical management. Orphalan's principal medicine is trientine tetrahydrochloride, a copper chelator intended to help remove excess copper in appropriate patients. The company reached an early regulatory milestone in March 2016, when the U.S. Food and Drug Administration granted orphan-drug designation to trientine tetrahydrochloride for Wilson's disease, excluding patients intolerant to penicillamine under the designation described in the available reference. In September 2017, the European Medicines Agency granted marketing authorization for the medicine under the Cuprior name. The authorization covered adults, adolescents, and children aged five years and older who were intolerant to D-penicillamine therapy. In October of the same year, Naseem Amin joined Orphalan as chief executive officer, providing the company with an identified senior leader during the period in which it moved from development toward commercialization. Commercial launch in the European Union took place in 2019. This established Orphalan as a commercial rare-disease company rather than solely a development-stage biotechnology business. The company later created the International Wilson Disease Registry in 2021. The registry is sponsored and managed by Orphalan and contains health data from consenting people with Wilson's disease in multiple countries. Its establishment reflects the company's involvement in disease-level data collection as well as medicine supply, although the available material does not describe the registry's full methodology, findings, or independent governance. The U.S. regulatory program advanced in April 2022. The FDA approved trientine tetrahydrochloride under the Cuvrior brand as a copper chelator for adult patients with stable Wilson's disease who were de-coppered and tolerant to penicillamine. The approval was associated with the CHELATE trial and included U.S. orphan-drug exclusivity. In September 2022, results from the CHELATE trial were published in The Lancet Gastroenterology & Hepatology. In November 2022, Shahzad Malik became chairman of the Orphalan board. Cuvrior launched commercially in the United States in April 2023. Orphalan then expanded into China: the National Medical Products Administration approved trientine tetrahydrochloride in December 2023 for adults, adolescents, and children aged five years and older who were intolerant to D-penicillamine therapy. The Chinese product is marketed as 科佩欧 (Ke Pei Ou), and its commercial launch was reported in March 2025. Across these stages, Orphalan's development has been defined by a focused rare-disease strategy, regulatory work in several major markets, and commercialization of the same active pharmaceutical ingredient under regionally adapted brand names. The available reference material states that the product is available in more than 30 countries. It does not establish a complete portfolio beyond trientine tetrahydrochloride, disclose the company's ownership or financial results, or identify additional current or former executives. Accordingly, Orphalan is best characterized from the available evidence as a privately held, internationally active specialist pharmaceutical company centered on Wilson's disease treatment.

1. 2025Chinese commercial launch

   科佩欧 launches commercially in China.

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2. 2023U.S. commercial launch

   Cuvrior becomes commercially available in the United States.

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3. 2023Chinese regulatory approval

   China's NMPA approves trientine tetrahydrochloride under the 科佩欧 brand for specified Wilson's disease patients.

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4. 2022U.S. approval of Cuvrior

   The FDA approves Cuvrior for specified adult patients with stable Wilson's disease and grants orphan-drug exclusivity.

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5. 2021International Wilson Disease Registry established

   Orphalan establishes a multinational registry for consenting people with Wilson's disease.

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6. 2019European commercial launch

   Trientine tetrahydrochloride launches commercially in the European Union as Cuprior.

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7. 2017European marketing authorization

   The EMA authorizes Cuprior for specified adults, adolescents, and children with Wilson's disease.

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8. 2016U.S. orphan-drug designation

   The FDA grants orphan-drug designation to trientine tetrahydrochloride for a Wilson's disease indication.

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9. 2011Orphalan is founded

   Orphalan S.A. is founded in France as an orphan-drug development and commercialization company.

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Specialist orphan-drug developer and commercializer focused on rare diseases, particularly Wilson's disease and therapies addressing abnormal copper accumulation.

• Trientine tetrahydrochlorideRare-disease medicine; copper chelator2019

  Orphalan's principal documented medicine is trientine tetrahydrochloride, a copper-chelating treatment for Wilson's disease. The product is marketed under different names by region: Cuprior in the European Union and certain other markets, Cuvrior in the United States, and 科佩欧 (Ke Pei Ou) in China. Its regulatory indications differ by jurisdiction but include specified adults, adolescents, and children aged five years and older, particularly patients who are intolerant to or otherwise meet the relevant treatment criteria concerning D-penicillamine. The product has been reported as available in more than 30 countries.

• 2023Expand trientine tetrahydrochloride into ChinaStrategy

  After European and U.S. regulatory progress, Orphalan pursued approval and commercialization of its Wilson's disease medicine in China.

  What changed. China's NMPA approved the product in December 2023 under the 科佩欧 brand, followed by commercial launch in March 2025.

  Aftermath. The China launch added another major international market to the product's documented geographic availability.

  Source
• 2022Pursue U.S. approval and commercialization of CuvriorProduct launch

  Orphalan sought U.S. approval for trientine tetrahydrochloride for adult patients with stable Wilson's disease who had been de-coppered and were tolerant to penicillamine.

  What changed. The FDA approved Cuvrior in April 2022 and granted orphan-drug exclusivity; the product launched commercially in the United States in April 2023.

  Aftermath. The decision expanded Orphalan's commercial reach into the U.S. market and was associated with evidence from the CHELATE trial.

  Source
• 2021Establish the International Wilson Disease RegistryStrategy

  Wilson's disease is rare, creating a need for broader international clinical and patient data beyond individual treatment settings.

  What changed. Orphalan sponsored and managed a centralized registry containing health data from consenting people with Wilson's disease in multiple countries.

  Aftermath. The registry expanded Orphalan's documented role in disease-focused data collection, although detailed outcomes are not provided in the reference material.

  Source
• 2017Proceed with European commercialization of CupriorProduct launch

  Following European marketing authorization for trientine tetrahydrochloride, Orphalan moved its principal product toward commercial availability for eligible Wilson's disease patients.

  What changed. The company launched Cuprior commercially in the European Union in 2019.

  Aftermath. The European launch established Orphalan's documented commercial presence in rare-disease medicines.

  Source

• 2025科佩欧 launches commercially in China

  Orphalan's trientine tetrahydrochloride product launched commercially in China under the 科佩欧 brand.

  Product launch
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• 2023Cuvrior launches commercially in the United States

  Orphalan launched its trientine tetrahydrochloride product commercially in the United States under the Cuvrior brand.

  Product launch
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• 2023China approves trientine tetrahydrochloride

  China's National Medical Products Administration approved trientine tetrahydrochloride, marketed as 科佩欧, for specified patients with Wilson's disease.

  Regulation
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• 2022FDA approves Cuvrior for eligible adults with stable Wilson's disease

  The FDA approved Cuvrior, trientine tetrahydrochloride, for adult patients with stable Wilson's disease who had been de-coppered and were tolerant to penicillamine. The product also received orphan-drug exclusivity.

  RegulationProduct launch
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• 2022CHELATE trial results published

  Results from the CHELATE trial supporting the U.S. regulatory history of Cuvrior were published in The Lancet Gastroenterology & Hepatology.

  Other
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• 2022Shahzad Malik appointed chairman

  Shahzad Malik was appointed chairman of the Orphalan board.

  Leadership change
  Source
• 2021International Wilson Disease Registry established

  Orphalan established a centralized registry containing health data from consenting people with Wilson's disease in countries around the world.

  Other
  Source
• 2019Cuprior launches commercially in Europe

  Orphalan began commercializing trientine tetrahydrochloride under the Cuprior brand in the European Union.

  Product launch
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• 2017EMA authorizes Cuprior for Wilson's disease

  The European Medicines Agency granted marketing authorization for trientine tetrahydrochloride, marketed as Cuprior, for adults, adolescents, and children aged five years and older who were intolerant to D-penicillamine therapy.

  RegulationProduct launch
  Source
• 2017Naseem Amin joins Orphalan as chief executive officer

  Naseem Amin joined the company as chief executive officer.

  Leadership change
  Source
• 2016FDA grants orphan-drug designation to trientine tetrahydrochloride

  The U.S. Food and Drug Administration granted orphan-drug designation to Orphalan's trientine tetrahydrochloride for Wilson's disease in the specified patient population.

  Regulation
  Source

1. Orphalan
2. Orphalan official website