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关注罕见病“真性红细胞增多症”：PTG-300获美国FDA孤儿药物认定

2020-06-18 Allan MedSci原创

真性红细胞增多症PTG-300
关注

真性红细胞增多症（polycythemia vera, PV）是一种骨髓增殖性肿瘤，骨髓增殖性肿瘤是一组源于造血干细胞、以形态学成熟度不一的骨髓细胞克隆增殖为特征的恶性肿瘤。

真性红细胞增多症（polycythemia vera, PV）是一种骨髓增殖性肿瘤，骨髓增殖性肿瘤是一组源于造血干细胞、以形态学成熟度不一的骨髓细胞克隆增殖为特征的恶性肿瘤。PV与其他MPN的区别在于红细胞量增多，且与血栓栓塞事件、白血病转化和/或骨髓纤维化的风险增加有关。

Protagonist制药公司今日宣布，美国食品药品监督管理局（FDA）已授予PTG-300治疗PV的孤儿药物认定。PTG-300是一种合成多肽，目前正在临床开发中。

Protagonist最近公布了真性红细胞增多症患者的II期临床试验的初步结果。Protagonist首席医学官医学博士Samuel Saks说道：“获得FDA孤儿药认证是Protagonist的一个重要里程碑。患有真性红细胞增多症的患者面临很高的疾病负担，PTG-300在治疗真性红细胞增多症方面具有新颖的治疗机制，已显示出良好的安全性”。

 

原始出处：

https://www.firstwordpharma.com/node/1733708?tsid=4

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    2020-09-11 ms6980045490879608

    #孤儿药物#

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    2020-06-20 kord1982

    #红细胞#

    0

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    2020-06-20 syscxl

    #罕见#

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    2020-06-20 10518098zz

    #美国FDA#

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    2020-06-20 linlin2317

    #红细胞增多症#

    0

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    2020-06-20 若水129

    #孤儿药物认定#

    0

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