EVENT DOSSIER
On September 8, 2026, Yicai reported that ten children suffering from extremely rare diseases were facing a serious crisis of lacking medication. Previously, these children had been in trouble due to the lack of effective treatment options, but recently there has been good news: the relevant medications have been successfully restored to supply and re-launched on the market, bringing new hope to the children. Currently, this situation is in a critical phase after the restoration of medication supply, and it is necessary to continue monitoring the specific subsequent impacts and the recovery status of the patients.
10名中国酪氨酸血症Ⅰ型患儿“救命药”尼替西农已在中国市场恢复供应。该药因加拿大生产商上游关键物料供应商被美国 FDA 检查发现缺陷导致全球性断供，企业经多轮沟通与论证后，于今年 4 月将中国列入首批恢复名单。目前患者用药已实现平稳过渡。此前断供期间，国内库存仅能维持约半年量，患儿家庭被迫压减药量或尝试“全球寻药”。尽管该药专利已过期且国内无本土仿制药企，但更换供应商、推动原研苏庇（Swedish Orphan Biovitrum AB）供货、寻找其他仿制药企代理及申请临床急需进口等四条替代路径均因合规成本、商业谈判僵局或政策限制而未能走通。目前进口成本较危机前明显上涨，企业处于亏本状态，且全球供应链脆弱性仍存隐忧。