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News & Updates

Stay informed about Orphalan's latest developments

From clinical breakthroughs to patient programmes and partnerships, discover what is new at Orphalan and in the rare disease community.

20 articles

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Orphalan announces European Medicines Agency (EMA) acceptance for review of Marketing Authorization Application (MAA) for KIZFIZO®
Company News

Orphalan announces European Medicines Agency (EMA) acceptance for review of Marketing Authorization Application (MAA) for KIZFIZO®

Marketing Authorization Application for KIZFIZO® accepted for review by the EMA for the treatment of malignant glioma.

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Orphalan announces European Medicines Agency (EMA) acceptance for review of Marketing Authorization Application (MAA) for KIZFIZO®
Company News

Orphalan announces European Medicines Agency (EMA) acceptance for review of Marketing Authorization Application (MAA) for KIZFIZO®

Marketing Authorization Application for KIZFIZO® accepted for review by the EMA for the treatment of malignant glioma.

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Rare Disease Run
Company News

Rare Disease Run

On International Rare Disease Day 2026, Orphalan joins the global movement with 43 colleagues across Europe, China, and the U.S. running for rare.

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Orphalan Announces the Acquisition of Orphelia Pharma
Press Release

Orphalan Announces the Acquisition of Orphelia Pharma

Strategic acquisition strengthens Orphalan’s position in rare disease therapeutics and expands portfolio capabilities.

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Orphalan Partners with MAP International and the Wilson Disease Association
Press Release

Orphalan Partners with MAP International and the Wilson Disease Association

Orphalan SA partners with MAP International and the Wilson Disease Association to donate Cuvrior® to patients with Wilson Disease in developing countries.

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Orphalan becomes the first European company to enter in China’s Wilson Disease market
Company News · Press Release

Orphalan becomes the first European company to enter in China’s Wilson Disease market

Strategic partnership with Kyuan Xinhai (Beijing) Medical Products Trade Co., Ltd to transform access to Wilson’s disease treatment for Chinese patients.

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Orphalan announces China NMPA’s approval of its trientine tetrahydrochloride product for the treatment of Wilson disease
Company News · Press Release

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

China’s National Medical Products Administration approves Orphalan’s trientine tetrahydrochloride product – the first trientine approved by the NMPA – for the treatment of Wilson disease.

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Tackling Rare Diseases: Wilson Disease Patient and Millions More Like Her Need Help
Company News

Tackling Rare Diseases: Wilson Disease Patient and Millions More Like Her Need Help

Over the years, I have been deeply moved by the stories of many patients, but one particularly stands out.

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Why We Should Care About Rare Disease Research – Life Science Leader
Company News

Why We Should Care About Rare Disease Research – Life Science Leader

Our CEO Naseem Amin authors an insightful article for Life Science Leader on why we should care about rare disease research.

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Orphalan announces US commercial launch of Cuvrior™ for the treatment of Wilson disease
Press Release

Orphalan announces US commercial launch of Cuvrior™ for the treatment of Wilson disease

Cuvrior™ now available in the US for the treatment of adult patients with stable Wilson disease who are de-coppered and tolerant to penicillamine.

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Podcast: Naseem Amin, Orphalan CEO, discusses Wilson’s Disease and the potential advancements in its treatments on DNA Today
Company News

Podcast: Naseem Amin, Orphalan CEO, discusses Wilson’s Disease and the potential advancements in its treatments on DNA Today

Orphalan CEO Naseem Amin sits down with DNA Today host Kira Dineen to discuss Wilson’s Disease and the potential advancements in its treatments.

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

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

Trial supported Orphalan’s successful application to the FDA for approval of Cuvrior™ (trientine tetrahydrochloride) for Wilson’s disease.

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

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

World’s first global Wilson’s Registry will provide an international platform for sharing real-world information about Wilson’s disease.

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Dr. Naseem Amin, Chief Executive Officer at Orphalan, discusses the announcement of the FDA approval of Cuvrior™
Company News · Video

Dr. Naseem Amin, Chief Executive Officer at Orphalan, discusses the announcement of the FDA approval of Cuvrior™

Dr. Naseem Amin, Chief Executive Officer at Orphalan, discusses the announcement of the FDA approval of Cuvrior™.

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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
Press Release

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

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Orphalan Speaks to Chair of Wilson’s Disease Patient Registry to Celebrate Rare Disease Day
Press Release

Orphalan Speaks to Chair of Wilson’s Disease Patient Registry to Celebrate Rare Disease Day

A Q&A between Omar Kamlin, Senior Medical Director at Orphalan SA, and Prof. Pramod Mistry, Director of Yale Lysosomal Disease Center, to mark World Rare Disease Day 2022.

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Orphalan strengthens its management team with two key appointments
Press Release

Orphalan strengthens its management team with two key appointments

Orphalan SA today announces two new appointments to the management team: Tim Jenkins as Chief Development Officer and Chris Homan as Chief Quality Officer.

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Orphalan announces FDA acceptance for filing of New Drug Application (NDA) for trientine tetrahydrochloride (TETA 4HCl) for the treatment of Wilson’s Disease
Press Release

Orphalan announces FDA acceptance for filing of New Drug Application (NDA) for trientine tetrahydrochloride (TETA 4HCl) for the treatment of Wilson’s Disease

NDA supported by positive data from Phase 3 CHELATE clinical trial. TETA 4-HCl previously granted Orphan Drug Designation for first-line treatment of Wilson’s Disease.

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Top line results of CHELATE trial presented at International Liver Congress 2021 — Top line Summary by Michael Schilsky, MD
Video

Top line results of CHELATE trial presented at International Liver Congress 2021 — Top line Summary by Michael Schilsky, MD

Prof. Michael Schilsky of Yale University presents top-line results from Orphalan’s CHELATE Phase 3 trial at EASL’s International Liver Congress 2021.

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This morning, Dr Weiss presented our Phase 3 data at @EASL | The Home of Hepatology’s International Liver Congress 2021.
Company News

This morning, Dr Weiss presented our Phase 3 data at @EASL | The Home of Hepatology’s International Liver Congress 2021.

Positive top-line data announced from our Phase 3 head-to-head trial of trientine tetrahydrochloride and d-penicillamine at EASL’s International Liver Congress 2021.

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Orphalan Announces Positive Top-Line Data with Trientine Tetrahydrochloride for Maintenance Patients with Wilson’s Disease
Press Release

Orphalan Announces Positive Top-Line Data with Trientine Tetrahydrochloride for Maintenance Patients with Wilson’s Disease

CHELATE trial confirmed trientine tetrahydrochloride was non-inferior to d-Penicillamine as maintenance therapy for Wilson’s Disease, with a markedly superior tolerability profile.

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