BioMarin· @BioMarin · X·· 2025-10-29精选AI 评分60
AI 导读
BioMarin宣布，美国FDA已受理其药品扩大适应症的补充生物制品许可申请（sBLA），并给予优先审评，拟将适应症扩展至患有PKU的青少年。PKU是一种罕见的遗传性代谢疾病，影响机体代谢苯丙氨酸（Phe）的能力。
推荐理由
FDA受理BioMarin扩大PKU适应症至青少年的sBLA并给予优先审评，读者可了解该罕见病治疗覆盖人群的拓展进展。
正文
The @US_FDA has accepted the supplemental Biologics License Application (sBLA) for Priority Review to expand our medicine’s indication to adolescents living with #PKU, a rare, inherited metabolic condition that affects the body’s ability to metabolize a part of a protein called phenylalanine (Phe).
Read more in our press release: https://bit.ly/4nprpcf
来源：BioMarin · x.com