FDA批准了赛诺菲的血友病药物-动脉网

FDA Approves Sanofi's Hemophilia Drug

benzinga 等信源发布 2025-03-31 08:28

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The U.S. Food and Drug Administration on Friday approved

美国食品和药物管理局周五批准了

Sanofi SA’s

赛诺菲集团的

SNY

索尼

Qfitlia (fitusiran), the first antithrombin-lowering (AT) therapy for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients (aged 12 or older) with hemophilia A or B with or without

Qfitlia（fitusiran），首个用于常规预防的抗凝血酶降低（AT）疗法，旨在预防或减少12岁及以上患有A型或B型血友病的成人和儿童患者的出血频率，无论是否具有抑制物。

factor VIII or IX inhibitors

因子VIII或IX抑制剂

。

The

approval

批准

is based on data from the ATLAS phase 3 studies that demonstrated clinically meaningful bleed protection as measured by annualized bleeding rates (ABR).

基于ATLAS三期研究的数据，该数据表明在年化出血率（ABR）方面具有临床意义的出血保护。

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By lowering AT, a protein that inhibits blood clotting, Qfitlia helps increase thrombin generation to restore hemostasis in hemophilia patients.

通过降低AT（一种抑制血液凝固的蛋白质），Qfitlia有助于增加凝血酶的生成，从而恢复血友病患者的止血功能。

In the ATLAS clinical development program, Qfitlia demonstrated low bleed rates across subgroups with as few as six injections a year. Key results include:

在ATLAS临床开发项目中，Qfitlia在各亚组中显示出极低的出血率，每年仅需六次注射。关键结果包括：

Significant bleed reduction by 71% in ABR for patients without inhibitors treated with Qfitlia prophylaxis compared to clotting factor concentrate on-demand and by 73% in ABR compared to bypassing agent on-demand for patients with inhibitors (estimated mean: ABR 5.1 vs. 19.1, respectively).

与按需使用凝血因子浓缩物相比，无抑制剂患者接受Qfitlia预防治疗后，ABR显著降低71%；与按需使用旁路制剂相比，有抑制剂患者的ABR降低73%（估计平均值：ABR分别为5.1和19.1）。

Median observed ABR during the open-label extension study was 3.8 in patients without inhibitors and 1.9 in patients with inhibitors.

在开放标签扩展研究中，未抑制剂患者的ABR中位观察值为3.8，而有抑制剂患者为1.9。

The median annualized spontaneous bleeding rate observed during the open-label extension study was 1.9 in patients without inhibitors and 1.9 in patients with inhibitors.

在开放标签扩展研究中观察到的中位年化自发性出血率在无抑制物的患者中为1.9，在有抑制物的患者中也为1.9。

Nearly half of the patients in the open-label extension study experienced one or fewer bleeds (31% zero bleeds and 47% 0-1 bleeds).

在开放标签扩展研究中，近一半的患者经历了一次或更少的出血（31% 无出血，47% 出血次数为0-1次）。

With the Qfitlia approval, the FDA cleared the Siemens Healthineers' INNOVANCE Antithrombin assay as a companion diagnostic for Qfitlia to measure AT levels.

随着Qfitlia获批，FDA批准西门子医疗的INNOVANCE抗凝血酶测定作为Qfitlia的伴随诊断，用于测量AT水平。

Qfitlia is a small interference RNA therapeutic that utilizes

Qfitlia 是一种利用小干扰 RNA 的治疗药物

Alnylam Pharmaceutical Inc.'s

阿尔尼拉姆制药公司

ALNY

阿尼利

ESC-GalNAc conjugate technology.

ESC-GalNAc结合技术。

Through the Qfitlia Testing Program with

通过Qfitlia测试计划

Labcorp

, the FDA-cleared companion diagnostic will be available to patients prescribed Qfitlia to measure AT levels at no cost.

，经FDA批准的伴随诊断将提供给被处方Qfitlia的患者，以免费测量AT水平。

A regulatory submission for Qfitlia for hemophilia A or B in adults and adolescents with or without inhibitors is under review in Brazil. A regulatory decision is expected in China in the second half of 2025.

巴西正在审查针对成人和青少年（无论是否具有抑制因子）的A型或B型血友病的Qfitlia监管提交。预计中国将在2025年下半年作出监管决定。

Price Action:

价格行为:

SNY stock is down 1.74% at $54.85 during the premarket session at the last check Monday.

SNY股票在周一最后一次检查时，盘前交易时段下跌1.74%，至54.85美元。

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Image by HJBC via Shutterstock.

图片由HJBC通过Shutterstock提供。

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