On January 5, 2024, the official website of the National Medical Products Administration (NMPA) announced that the Class 5.1 new drug Trientine Hydrochloride Tablets submitted by Orphalan have been officially approved for marketing, for the treatment of hepato-lenticular degeneration (Wilson's disease) in penicillamine-intolerant children aged ≥5 years and adults. This is a rare autosomal recessive hereditary copper metabolism disorder, which may be life-threatening due to impaired hepatic copper transport, and has been included in China's "First List of Rare Diseases" in May 2018. From the submission of the marketing application in China in September 2022 to the official approval at the end of December 2023, R&G, as an industry-leading CRO, provided NDA services such as regulatory strategy, application dossier preparation and submission, and communication with regulatory authorities for this product. It successfully obtained priority review and approval, as well as clinical trial exemption for marketing approval, helping Orphalan's product become the first approved trientine formulation in China.
Screenshot of CDE Priority Review Announcement Image Source: CDE Official Website
Hepato-lenticular degeneration, also known as Wilson's disease, is an autosomal recessive hereditary disease characterized by copper metabolism disorders, and currently has no effective treatments. People with this disease are called "copper children". This disease causes disorders in the transport and excretion of copper ions in the human body and can easily lead to liver and brain damage; patients need lifelong medication.
According to reports, this product is the first trientine formulation approved for marketing in China. Trientine Hydrochloride is a copper ion chelator that can eliminate absorbed copper in the body by forming stable complexes, acting similarly to penicillamine, and can promote the excretion of copper through the kidneys, reducing copper absorption. Trientine can replace penicillamine in cases of penicillamine intolerance, and trientine has a better ability to cross the blood-brain barrier than existing therapeutic drugs, showing advantages for patients with cerebral phenotype. This time, it was exempted from clinical trials and received priority approval, accelerating the product's launch in China, bringing better treatment options to adult and pediatric patients with Wilson's disease.
Screenshot of Product Approval for Marketing. Image Source: National Medical Products Administration website
During the process of assisting the successful NDA submission of Trientine Hydrochloride Tablets, the R&G registration team closely collaborated with the Orphalan team, formulated a rigorous submission plan, and ensured the smooth progress of the project in multiple aspects, providing crucial and strong support for the successful clinical trial exemption of Trientine Hydrochloride Tablets. The professional capabilities of the R&G registration team have also been highly recognized by the Orphalan team.
At present, the R&G Regulatory Affairs Department has experienced experts and teams, having accumulated experience in nearly 300 domestic and international registration projects, with in-depth research and understanding of domestic and international registration-related regulations. Relying on the team's rich experience and professional capabilities, they can provide full lifecycle registration services for new drug clinical development and post-marketing for global pharmaceutical R&D enterprises. In the future, R&G will continue to assist in the global research, development, registration, and marketing of innovative drugs, so that more innovative drugs and quality therapies can be approved and marketed as soon as possible, benefiting patients.
