江苏恒瑞医药股份有限公司 关于获得美国FDA孤儿药资格认定的公告

Core Viewpoint - Jiangsu Hengrui Medicine Co., Ltd. has received orphan drug designation from the U.S. FDA for its product, injection of Rukang Trastuzumab in combination with Atezolizumab and chemotherapy for the treatment of gastric cancer or gastroesophageal junction adenocarcinoma, which will provide opportunities for policy support in product development, registration, and commercialization [1][4]. Group 1: Drug Information - Drug Name: Injection of Rukang Trastuzumab [1] - Indication: Used in combination with Atezolizumab and chemotherapy for gastric cancer or gastroesophageal junction adenocarcinoma [1] - Application Number: DRU-2025-10850 [1] - Approval Conclusion: Granted orphan drug status under Section 526 of the Federal Food, Drug, and Cosmetic Act [1]. Group 2: Market Context - In 2022, gastric cancer ranked 5th in global cancer incidence and mortality, with 968,400 new cases and 659,900 deaths worldwide [2]. - In China, there were 358,700 new cases and 260,400 deaths, ranking 5th in incidence and 3rd in mortality [2]. - Current first-line treatment standards have shown some clinical effectiveness but still face unmet clinical needs due to short survival times and poor prognosis [2]. Group 3: Drug Mechanism and Competition - Rukang Trastuzumab binds to HER2-expressing tumor cells, inducing apoptosis through a mechanism involving toxin release in lysosomes [3]. - The drug was approved for use in China in May 2025 for adult patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) who have previously received at least one systemic treatment [3]. - Competing products include Ado-trastuzumab emtansine (Kadcyla) and Fam-trastuzumab deruxtecan (Enhertu), with combined global sales projected at approximately $6.557 billion for 2024 [3]. Group 4: Impact of Orphan Drug Designation - The orphan drug designation will expedite clinical trial and registration processes [4]. - The company will benefit from policy support, including tax credits for clinical trial costs, waiver of new drug application fees, and seven years of market exclusivity post-approval [4].