美国林业发展局接受了对患有罕见的免疫障碍APDS的4至11岁儿童的Leniolisib申请,决定应于2026年1月31日做出。 U.S. FDA accepted leniolisib application for children 4–11 with rare immune disorder APDS, with decision due Jan. 31, 2026.

美国林业发展局已接受制药集团对口服PI3K抑制剂Leniolisib的补充药物应用,针对4岁至11岁儿童的PI3K综合症(APDS),这是一种罕见的免疫障碍。 The U.S. FDA has accepted Pharming Group’s supplemental drug application for leniolisib, an oral PI3Kδ inhibitor, for children aged 4 to 11 with activated PI3Kδ syndrome (APDS), a rare immune disorder. 该申请得到了优先审查,预计将在2026年1月31日前作出决定。 The application received Priority Review, with a decision expected by January 31, 2026. 第三阶段试验的数据表明,在12周内,加上8个月的安全数据,疾病标记有所改善,淋巴发病率下降,幼稚B细胞增加。 Data from a Phase III trial showed improved disease markers—reduced lymphadenopathy and increased naïve B cells—within 12 weeks, along with eight months of safety data. 如果得到批准,Leniolisib将成为全球使用APDS治疗12岁以下儿童的第一个治疗办法。 If approved, leniolisib would be the first treatment for children under 12 with APDS globally. 其他几个国家正在审查已经为这12岁及12岁以上的人核准的该药物。 The drug, already approved for those 12 and older, is under review in several other countries. 由基因突变引起的APDS导致经常感染、免疫机能丧失和长期并发症,如淋巴瘤和肺损伤,往往是在症状开始多年后诊断出来。 APDS, caused by genetic mutations, leads to recurrent infections, immune dysfunction, and long-term complications like lymphoma and lung damage, often diagnosed years after symptoms begin.