Orphalan

Orphalan
TypePrivate
IndustryBiotechnology
Founded2011
HeadquartersParis, France
Key people
Naseem Amin (Chief Executive Officer) Shahzad Malik (Chairman of the Board)
ProductsTrientine tetrahydrochloride/盐酸曲恩汀 (Cuprior/Cuvrior/科佩欧)
Number of employees
125
Websitewww.orphalan.com

Orphalan S.A. is an orphan drug development and commercialization company headquartered in Paris, France. It specializes in the development of treatments for patients with Rare diseases.[1][2]

Its trientine tetrahydrochloride (盐酸曲恩汀) product, (branded as Cuprior in the EU[3] and some key markets, Cuvrior in the USA[4] and 科佩欧 (Ke Pei Ou) in China[5]) is a treatment for Wilson's disease, which is available in over 30 countries.[2]

History

Orphalan S.A. was founded in 2011.[2] In March 2016, trientine tetrahydrochloride (brand name Cuvrior in the USA) was granted orphan drug designation in the USA by the Food and Drug Administration (FDA) for the indication: treatment of Wilson's disease excluding patients intolerant to penicillamine.[6]

In September 2017, the European Medicines Agency (EMA) granted marketing authorization for trientine tetrahydrochloride (brand name Cuprior)[7] for the treatment of Wilson's disease in adults, adolescents and children ≥5 years intolerant to D-penicillamine therapy.[3]

In October 2017, Naseem Amin joined Orphalan as Chief Executive Officer.

In 2019, trientine tetrahydrochloride was launched commercially in the EU[8]

In 2021, the International Wilson Disease (iWD) Registry was established (a centralized database that is sponsored and managed by Orphalan SA).[9][1] This includes health data from consenting people with Wilson's disease from various countries around the world.[9][1]

In April 2022, trientine tetrahydrochloride (brand name Cuvrior in the USA) was approved by the FDA and granted Orphan Drug Exclusivity (ODE).[10] as a copper chelator indicated for the treatment of adult patients with stable Wilson's disease who are de-coppered and tolerant to penicillamine, based on results from the CHELATE trial[10]

In September 2022, results from the CHELATE trial were published in The Lancet Gastroenterology & Hepatology.[11][12]

In November 2022, Shahzad Malik was appointed Chairman of the Orphalan Board.

In April 2023, trientine tetrahydrochloride was launched commercially in the USA.[8]

In December 2023, China's National Medical Products Administration (NMPA) approved trientine tetrahydrochloride (盐酸曲恩汀, brand name 科佩欧) for the treatment of Wilson's disease in adults, adolescents and children ≥5 years intolerant to D-penicillamine therapy.[5][13][14]

In March 2025, trientine tetrahydrochloride was launched commercially in China.[5]

Products

Trientine tetrahydrochloride, a copper-chelating agent for the treatment of Wilson's disease.[3][4]

References

  1. ^ a b c "Our Work". Orphalan SA. Retrieved 2025-11-13.
  2. ^ a b c "About Us". Orphalan SA. Retrieved 2025-11-13.
  3. ^ a b c "Cuprior | European Medicines Agency (EMA)". www.ema.europa.eu. 2017-09-05. Retrieved 2025-11-13.
  4. ^ a b "DailyMed - CUVRIOR- trientine tetrahydrochloride tablet, film coated". dailymed.nlm.nih.gov. Retrieved 2025-11-27.
  5. ^ a b c Kothari, Minesh (2025-03-07). "Orphalan becomes the first European company to enter in China's Wilson Disease market". Orphalan SA. Retrieved 2025-11-13.
  6. ^ "Search Orphan Drug Designations and Approvals". www.accessdata.fda.gov. Archived from the original on 2025-04-16. Retrieved 2025-11-13.
  7. ^ "Cuprior | European Medicines Agency (EMA)". www.ema.europa.eu. 2017-09-05. Retrieved 2025-11-27.
  8. ^ a b admin (2023-04-20). "Orphalan announces US commercial launch of Cuvrior™ for the treatment of Wilson disease". Orphalan SA. Retrieved 2025-11-13.
  9. ^ a b "Home". Wilson's Disease Registry. Retrieved 2025-11-13.
  10. ^ a b admin (2022-05-02). "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". Orphalan SA. Retrieved 2025-11-27.
  11. ^ Schilsky, Michael L.; Czlonkowska, Anna; Zuin, Massimo; Cassiman, David; Twardowschy, Carlos; Poujois, Aurelia; Gondim, Francisco de Assis A.; Denk, Gerald; Cury, Rubens G.; Ott, Peter; Moore, Joanna; Ala, Aftab; D'Inca, Renata; Couchonnal-Bedoya, Eduardo; D'Hollander, Koenraad (December 2022). "Trientine tetrahydrochloride versus penicillamine for maintenance therapy in Wilson disease (CHELATE): a randomised, open-label, non-inferiority, phase 3 trial". The Lancet. Gastroenterology & Hepatology. 7 (12): 1092–1102. doi:10.1016/S2468-1253(22)00270-9. ISSN 2468-1253. PMID 36183738.
  12. ^ Kothari, Minesh (2022-09-30). "Orphalan announces publication of results from the CHELATE trial in The Lancet Gastroenterology & Hepatology". Orphalan SA. Retrieved 2025-11-27.
  13. ^ admin (2024-01-18). "Orphalan announces China NMPA's approval of its trientine tetrahydrochloride product for the treatment of Wilson disease". Orphalan SA. Retrieved 2025-11-27.
  14. ^ "国家药品监督管理局". www.nmpa.gov.cn. Archived from the original on 2025-06-12. Retrieved 2025-11-27.

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