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 ("iWDRegistry")

• 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 [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.

The registry was established by Orphalan to provide physicians from around the world with a platform to engage, share cases and consult with each other on this rare disease. iWD Registry intends to describe the natural history of treated Wilson's Disease patients and to explore the clinical phenotype, geographic, treatment, and gender influences on the course of the condition. The registry will also encourage the identification of new standards for the management of Wilson's Disease, provide long-term data for epidemiological research and evaluate adherence to therapies. The first five patients have been enrolled at the Children's Memorial Health Institute, and the Institute of Psychiatry and Neurology, Warsaw. Further sites in Belgium, Spain, Germany, the UK, and France will open to recruitment in the coming months.

Upon completion, iWD Registry is expected to include 500 individuals. Wilson's Disease patients over the age of 12 who provide informed consent are eligible for inclusion, with monitoring taking place during their regular follow-up visits for ongoing care by their primary physician.

Wilson's disease is a rare inherited disorder of copper transport, primarily affecting the liver and brain, which can be fatal if left untreated. Orphalan commercializes Cuprior, its trientine tetrahydrochloride product for the treatment of Wilson's disease in Europe, and is launching Cuvrior, recently approved by the FDA, in the US in Q4 this year.

Dr. Omar Kamlin, Medical Director at Orphalan, commented: "The enrolment of the first five people in the iWD Registry further highlights Orphalan's commitment to supporting patients affected by this disease. Much of the evidence that has so far been used to develop Wilson's Disease treatment guidelines is based on retrospective analyses of data from single sites. By including prospective data from patients worldwide, this registry will help physicians collaborate to find better standards for the treatment of Wilson's Disease. My congratulations go to Prof Socha, Dr. Janczyk, and their team at the Children's Memorial Health Institute in Warsaw for enrolling the very first patient."

Professor Pramod Mistry, MBBS, PhD, MA, MD, Director of Yale Lysosomal Disease Centre,added: "An international registry of Wilson's Disease patients is long overdue, and I congratulate the team at Orphalan for establishing it. Wilson's Disease is a global condition and it's not uncommon for me to receive an enquiry on management from a physician in, say, India. The more we share information and cases, the better we'll be able to support patients. The registry will also harness patient participation because there will always be questions from patients that individual centres cannot answer. I believe we'll see a big positive impact on patient care."

-ENDS-

About Orphalan

Orphalan is a pioneering, international orphan drug development and commercialization company. Founded in 2011, the company develops and delivers innovative therapies for people living with orphan diseases, and is initially focused on Wilson's Disease, a rare genetic disorder that can be life-threatening if untreated. Orphalan commercializes Cuprior, its trientine tetrahydrochloride product for the treatment of Wilson's disease in Europe, and is launching Cuvrior, recently approved by the FDA, in the US this year. For more information visit www.orphalan.comand follow us on LinkedIn.

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Consilium Strategic Communications:

Mary-Jane Elliott, Tracy Cheung, Davide Salvi

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