需补充儿童患者临床试验相关数据 天坛生物一款血友病药物撤回上市申请

Core Viewpoint - Tian Tan Biological has withdrawn its application for a hemophilia drug after 10 months of acceptance, citing the need for additional clinical trial data for children under 12 years old as per the CDE's review [1][4]. Group 1: Company Developments - Tian Tan Biological's drug, "Recombinant Human Coagulation Factor VIIa," had an investment of 266 million yuan in R&D and was expected to treat bleeding in patients aged 12 and older [1][3]. - The company’s product was designed to be similar to the imported drug Novoseven, which has been in the market for over 20 years without any domestic alternatives until recently [2][3]. - The recent announcement did not specify age groups for the drug's indications, aligning it closely with the imported product [4]. Group 2: Market Context - The hemophilia drug market is predominantly controlled by foreign companies, with over 90% market share, highlighting the challenges faced by domestic firms [7]. - The approval of the first domestic recombinant factor VIIa by Zhengda Tianqing in July 2023 marked a significant milestone, although its indications are limited compared to Novoseven [2][6]. - The domestic market for rare disease drugs is evolving, with 21 rare disease drugs approved in the first half of 2025, indicating a shift towards addressing previously unmet needs [7][8]. Group 3: Regulatory and Industry Trends - Regulatory bodies are increasingly focused on ensuring that new drugs address comprehensive patient needs, particularly for children, as seen in the requirement for additional pediatric data for Tian Tan's drug [6]. - The Chinese government has initiated programs to support the development of pediatric drugs and streamline approval processes, which may enhance the availability of treatments for rare diseases [7]. - The competitive landscape is changing, with domestic companies pushing for market share, leading to price reductions in existing treatments, thereby improving accessibility for patients [8].