Ultragenyx Pharmaceutical Inc宣布，其针对Ia型糖原贮积症（GSDIa）的AAV基因疗法DTX401的生物制剂许可申请（BLA），已获得美国食品药品监督管理局（FDA）的受理并被授予优先审评资格。
此项决定标志着DTX401在迈向商业化应用的道路上取得了关键性监管进展。优先审评资格的授予，通常意味着FDA认为该疗法有潜力在治疗、诊断或预防严重疾病的安全性或有效性方面提供显著改进。
这一里程碑将为患有此种罕见遗传性代谢疾病的患者带来新的治疗希望。
Ultragenyx Pharmaceutical Inc宣布，其针对Ia型糖原贮积症（GSDIa）的AAV基因疗法DTX401的生物制剂许可申请（BLA），已获得美国食品药品监督管理局（FDA）的受理并被授予优先审评资格。
此项决定标志着DTX401在迈向商业化应用的道路上取得了关键性监管进展。优先审评资格的授予，通常意味着FDA认为该疗法有潜力在治疗、诊断或预防严重疾病的安全性或有效性方面提供显著改进。
这一里程碑将为患有此种罕见遗传性代谢疾病的患者带来新的治疗希望。
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