Xin Lang Cai Jing·2025-12-26 12:21Core Viewpoint - AstraZeneca announced the approval of Ipupronit Sodium Injection (brand name Wainua) in China for the treatment of adult hereditary transthyretin amyloidosis polyneuropathy (ATTRv-PN), marking a significant milestone as it is the only gene-silencing agent approved for this condition in the country [1][6]. Group 1: Approval and Treatment Benefits - The approval is based on positive results from the NEURO-TTRansform Phase III clinical study, which demonstrated consistent and sustained clinical benefits in patients treated with Ipupronit Sodium compared to a placebo over a 66-week observation period [3][8]. - Key endpoints showed improvements in serum transthyretin (TTR) levels and neuropathy impairment scores, as well as quality of life assessments, indicating the drug's effectiveness [3][8]. Group 2: Disease Context and Treatment Mechanism - ATTRv-PN is a debilitating disease that can lead to peripheral nerve damage, with patients typically experiencing motor function impairment within five years of diagnosis and an average life expectancy of ten years without treatment [4][9]. - Ipupronit Sodium is administered monthly via an automatic injector, enhancing medication convenience and potentially improving patients' quality of life [1][6]. - The drug works by upstream inhibition of TTR production in the liver, making it a promising treatment for various types of transthyretin amyloidosis [4][9]. Group 3: Global Development and Ongoing Research - Ipupronit Sodium has also been approved in the United States and the European Union, with ongoing efforts to secure approvals in more countries [5][9]. - The drug is currently undergoing the CARDIO-TTRansform Phase III clinical study to evaluate its efficacy in treating adult transthyretin cardiac amyloidosis (ATTR-CM), which is the largest clinical study of its kind with over 1,400 participants [5][9].