Ultragenyx Pharmaceutical（纳斯达克代码：RARE）已顺利完成向美国食品药品监督管理局滚动提交DTX401 AAV基因疗法治疗Ia型糖原贮积症的生物制品许可申请。这一里程碑标志着该创新疗法在监管审批道路上迈出关键一步，为罕见病患者带来新的治疗希望。
DTX401作为针对GSDIa的基因治疗产品，采用腺相关病毒载体技术，旨在通过单次给药实现长期疗效。滚动提交策略允许药企在完成不同申报模块后分阶段递交资料，既能加速审评进程，又能确保数据的完整性与严谨性。
此次申报基于多项临床研究数据，结果显示DTX401能显著改善患者的代谢控制水平，同时展现出良好的安全性与耐受性。若最终获批，该疗法将填补GSDIa领域精准治疗的空白，重塑罕见病治疗格局。
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