Actemra获准用于治疗多关节青少年特发性关节炎

圣路易斯(MD Consult)——2013年4月30日，基因泰克宣布美国食品药品管理局(FDA)已批准Actemra(托珠单抗)——一种人源化白介素6受体拮抗剂——用于年龄≥2岁、处于疾病活动期的多关节青少年特发性关节炎(PJIA)患儿。Actemra可以单独使用，亦可与甲氨蝶呤(MTX)联用。

这项FDA批准令标志着Actemra在儿童中获得了第二项适应证。该药还获准用于治疗年龄≥2岁儿童的活动性系统性特发性关节炎。除了儿童以外，Actemra还可用于对至少1种缓解疾病抗风湿药物应答不充分的中至重度活动性类风湿性关节炎(RA)成人患者的治疗。

本次扩大适应证是基于1项针对PJIA患儿的3期研究数据。该研究设置了1个开放标记期，之后是1个随机、双盲、安慰剂对照撤药期。接受Actemra治疗者产生了有临床意义的PJIA症状和体征的改善。在随机、双盲、安慰剂对照撤药期中，接受Actemra治疗者的疾病发作次数显著少于接受安慰剂治疗者(26% vs. 48%)。

迄今采集到的关于PJIA患者使用Actemra的安全性数据，与既往纳入接受Actemra治疗者的研究观察到的结果一致。在上文所述的3期研究中，感染是40周内最常见的不良事件，也是最常见的严重不良事件。已知与使用Actemra相关的实验室指标异常在本项研究中也有所显现，包括白细胞计数和血小板计数下降，丙氨酸转氨酶和天冬氨酸转氨酶水平升高。

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ST LOUIS (MD Consult) - On April 30, 2013, Genentech announced that the US Food and Drug Administration (FDA) has approved Actemra (tocilizumab), a humanized interleukin-6 receptor antagonist, for the treatment of polyarticular juvenile idiopathic arthritis (PJIA) in children aged 2 years and older with active disease. Actemra can be given alone or in combination with methotrexate (MTX).

This FDA approval marks the second Actemra indication in children. The drug is also approved for the treatment of active systemic juvenile idiopathic arthritis in patients aged 2 years and older. In addition to the indications in children, Actemra is also approved for the treatment of adult patients with moderately to severely active rheumatoid arthritis (RA) who have shown an inadequate response to one or more disease-modifying antirheumatic drugs.

This expanded indication for Actemra was granted on the basis of data from a phase 3 study in children with PJIA, which featured an open-label phase, followed by a randomized, double-blind, placebo-controlled withdrawal phase. Patients treated with Actemra experienced clinically meaningful improvement in signs and symptoms of PJIA. In the randomized, double-blind, placebo-controlled withdrawal phase of the trial, Actemra-treated patients experienced significantly fewer disease flares compared with placebo-treated patients (26% vs 48%).

Safety data collected to date regarding the use of Actemra in patients with PJIA is consistent with that observed in previous studies that included Actemra-treated patients. In the aforementioned phase 3 study, infections were the most common adverse events and serious adverse events over 40 weeks. Laboratory abnormalities known to occur with the use of Actemra were also observed in this study, including decreased white blood cell and platelet counts, and elevated levels of alanine aminotransferase and aspartate aminotransferase.

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