获受理10个多月后，天坛生物（SH600161，股价17.61元，市值348.22亿元）旗下一款投入2.66亿元研 发的血友病药物，撤回了上市申请。 11月19日晚间，这家国内头部血液制品公司发布公告，称下属企业成都蓉生药业有限责任公司（以下简 称成都蓉生）撤回了"注射用重组人凝血因子Ⅶa"的药品注册申请，原因是根据CDE（国家药品监督管 理局药品审评中心）的审评意见，需要补充儿童（＜12岁）患者群体的临床试验相关数据。 《每日经济新闻》记者注意到，目前国内共有2款同类药物获批，分别来自丹麦药企诺和诺德和国内药 企正大天晴药业集团南京顺欣制药有限公司（以下简称正大天晴），后者的药物于今年7月获批，适用 于符合条件的成人及青少年（12岁以上）先天性血友病患者的出血治疗。 那么，问题来了：同类国产药物的获批适应证不包含12岁以下患者，成都蓉生为何需要补充儿童数据？ 被外资企业占有九成以上份额的罕见病儿童药物市场，正在发生哪些变化？ 唯一国产药物7月获批 要了解注射用重组人凝血因子Ⅶa这款药物，首先要了解血友病。 在相关资料中，正大天晴表示该药物实现了本土化生产，打破了进口依赖，解决了进口rFⅦa供应短缺 的现状。 ...

Core Viewpoint - Tian Tan Bio's application for a hemophilia drug was withdrawn after 10 months of acceptance due to the need for additional clinical trial data for children under 12 years old, highlighting regulatory scrutiny in the rare disease market [2][11]. Company Summary - Tian Tan Bio's subsidiary Chengdu Rongsheng Pharmaceutical withdrew the registration application for "Recombinant Human Coagulation Factor VIIa Injection," which had a research investment of 266 million yuan [2][5]. - The drug's indication was aligned with that of imported products, focusing on adult and adolescent patients, but lacked data for younger patients, which is now a regulatory requirement [4][11]. - The company had previously completed a Phase III clinical trial, but the latest announcement did not specify age groups for the patient population [4][5]. Industry Summary - The hemophilia drug market in China is dominated by foreign companies, with over 90% market share, and there is a growing demand for domestic alternatives [12][13]. - The recent approval of a similar drug by Zhengda Tianqing, which does not cover children under 12, indicates a gap in the market that domestic companies are trying to fill [8][11]. - Regulatory bodies are pushing for comprehensive data that includes pediatric populations, reflecting a shift towards more inclusive treatment options for rare diseases [11][12]. - The domestic pharmaceutical industry is accelerating its efforts in rare disease drug development, with 21 rare disease drugs approved in the first half of 2025, indicating a positive trend towards addressing unmet medical needs [12][13].