Orphalan announces China NMPA’s approval of its trientine tetrahydrochloride product for the treatme

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 National Medical Products Administration (NMPA). This product, already marketed as Cuprior® in the EU, the UK, Saudi Arabia, Switzerland, and Colombia, and as Cuvrior® in the US, represents the first trientine approved by the NMPA. It offers a new treatment option for Wilson disease in children aged five years and older and adults intolerant to penicillamine therapy.

China is home to an estimated 80,000 individuals with Wilson disease, of whom approximately half have their diagnosis confirmed, and 25,000 are receiving regular treatment. The introduction of new treatment alternatives such as Orphalan’s trientine tetrahydrochloride, particularly for patients who are intolerant to currently available therapies, presents a significant opportunity to meet the needs of a broader segment of the Wilson disease population in China. Orphalan anticipates its product to be available in the country in the coming months.

Dr Naseem Amin, Chief Executive Officer at Orphalan, said: “We are pleased with the marketing authorisation approval of our trientine tetrahydrochloride product in China, which offers a well-tolerated and effective treatment option for patients with Wilson disease. This approval is a crucial step towards providing a much-needed treatment option for the Wilson disease community in China and reaffirms our commitment to improving the experience for patients living with rare diseases worldwide.”

Yan Qin, Director of Wuhan Tongxin (Wilson disease patient group in China) added: “We were thrilled when we heard that NMPA approved Orphalan's trientine tetrahydrochloride in China. It is a significant step for those of us living with Wilson disease, representing a much-needed alternative treatment option. This approval, a result of Orphalan's patient-focused approach, offers us a chance to better manage our condition with a new, effective treatment.”

Wilson disease is a rare inherited disorder of copper transport primarily affecting the liver and brain. Early treatment with legacy agents such as D-penicillamine (DPA), trientine HCI, and zinc salts can effectively slow the progression of the disease, however these options may not be suitable for long-term care due to adverse events associated with DPA and the need for close monitoring during treatment with zinc salts and DPA.

        ENDS        

About Orphalan

Orphalan is an international orphan drug development and commercialization company headquartered in Paris. Founded in 2011, the Company develops and deliver innovative therapies for people living with rare and debilitating diseases. Our trientine tetrahydrochloride product has been approved for the treatment of Wilson disease and is available in more than 20 countries, branded as Cuprior® in EU, the UK, Saudi Arabia, Switzerland and Columbia, and as Cuvrior® in the United States. For more information visit www.orphalan.com and follow us on LinkedIn.

For more information, please contact:

Orphalan:

Géraldine van den Broek, Head of Corporate & BD

Tel: +33 (0)1 42 49 82 64

info@orphalan.com

ICR Consilium:

Tracy Cheung, Sukaina Virji, Davide Salvi

Tel: +44 (0) 203 709 5700

orphalan@consilium-comms.com