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At Orphalan we identify, develop and provide access to innovative treatments for patients with rare diseases.

Latest News, Press Releases & Videos

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

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

Paris, France 18 January, 2024 – Orphalan SA (“Orphalan” or “the Company”), an international orphan drug development and commercialization company, today announces the approval of its trientine tetrahydrochloride product by China’s…
January 18, 2024
Tackling rare diseases: Wilson disease patient and millions more like her need help
Tackling rare diseases: Wilson disease patient and millions more like her need helpCompany News

Tackling rare diseases: Wilson disease patient and millions more like her need help

Over the years, I’ve been deeply moved by the stories of many patients, but one particularly stands out. A woman talked about her teenage daughter’s Wilson disease — a rare…
September 25, 2023
Why We Should Care About Rare Disease Research – Life Science Leader
Why We Should Care About Rare Disease Research – Life Science LeaderCompany News

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

Why We Should Care About Rare Disease Research “Access to life-saving or life-enhancing treatments should not be determined by the rarity of the disease; on the contrary, our greater understanding…
May 9, 2023
Orphalan announces US commercial launch of Cuvrior™ for the treatment of Wilson disease
Orphalan announces US commercial launch of Cuvrior™ for the treatment of Wilson diseasePress 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 - Oral administration offers dosing flexibility…
April 20, 2023
Podcast: Naseem Amin, Orphalan CEO, discusses Wilson’s Disease and the potential advancements in its treatments on DNA Today
Podcast: Naseem Amin, Orphalan CEO, discusses Wilson’s Disease and the potential advancements in its treatments on DNA TodayCompany News

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

DNA Today is a multi-award-winning podcast and radio show exploring how genetics impacts our health. Host/Producer Kira Dineen interviews leaders in genetics including genetic counsellors, researchers, physicians, and patient advocates…
February 6, 2023
Orphalan announces publication of results from the CHELATE trial in The Lancet Gastroenterology & Hepatology
Orphalan announces publication of results from the CHELATE trial in The Lancet Gastroenterology & HepatologyPress Release

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

Trial supported Orphalan’s successful application to the United States Food and Drug Administration (FDA) for approval of Cuvrior™ (trientine tetrahydrochloride) for Wilson’s disease Paris, France 30 September, 2022 – Orphalan…
September 30, 2022
Orphalan announces enrolment of the first five patients in the International Wilson’s Disease Patient Registry (“iWD Registry”)
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 Registry expected to include 500 individuals affected by this rare disease Paris, France…
August 8, 2022
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™Company NewsVideo

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™ in this video: https://www.youtube.com/watch?v=Yu2WsUwtayE
May 3, 2022
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 penicillaminePress 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. Paris, France 2 May 2022 – Orphalan…
May 2, 2022
Orphalan Speaks to Chair of Wilson’s Disease Patient Registry to Celebrate Rare Disease Day
Orphalan Speaks to Chair of Wilson’s Disease Patient Registry to Celebrate Rare Disease DayPress Release

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

Conversation between Omar Kamlin, Senior Medical Director at Orphalan SA and Prof Pramod Mistry, MBBS, PhD, MA, MD, Director of Yale Lysosomal Disease Center and Gaucher Disease Treatment Center at…
February 28, 2022
Orphalan strengthens its management team with two key appointments
Orphalan strengthens its management team with two key appointmentsPress Release

Orphalan strengthens its management team with two key appointments

Paris, France 1 February 2022 – Orphalan SA, a Company that identifies, develops, and delivers therapies worldwide for orphan diseases, today announces two new appointments to the management team. Tim…
February 1, 2022
FDA acceptance for filing of New Drug Application (NDA) for trientine tetrahydrochloride (TETA 4HCl) for the treatment of Wilson’s Disease
FDA acceptance for filing of New Drug Application (NDA) for trientine tetrahydrochloride (TETA 4HCl) for the treatment of Wilson’s DiseasePress Release

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

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…
September 2, 2021
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We believe that patients across the world, with a rare disease, should have access to innovative treatment that improves their care and positively impacts their lives and those of their loved ones.

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