FDA批准激酶抑制剂Pimjetmi治疗腱鞘巨细胞瘤 推荐剂量每日一次50毫克
华盛顿 — 美国食品药品管理局（FDA）11月10日批准 pimicotinib（商品名 Pimjetmi，EMD Serono 公司）上市。这是一种激酶抑制剂，用于成人有症状的腱鞘巨细胞瘤（TGCT）患者，适用情形是手术切除可能导致功能受限加重或严重并发症。
这项批准的疗效依据是 MANEUVER 试验（NCT05804045）。这是一项双盲、随机（2比1）、安慰剂对照的国际试验，受试者为确诊有症状腱鞘巨细胞瘤、病灶可测量且至少一处病灶不小于2厘米、手术可能造成活动障碍或严重并发症的患者，按地区（中国与非中国）分层后，随机接受每日一次 pimicotinib 50毫克或安慰剂。
FDA 给出的推荐剂量为每日一次口服50毫克，可与食物同服、也可空腹服用。该药此前已获得快速通道与突破性疗法认定。
安全性方面，最常见的不良反应（发生率超过20%）包括瘙痒、皮疹、面部水肿、眼眶周围水肿、疲劳、恶心、头痛与外周水肿；最常见的实验室异常（超过30%）包括肌酸磷酸激酶升高、乳酸脱氢酶升高、天冬氨酸氨基转移酶升高、脂肪酶升高、胆固醇升高、淀粉酶升高与丙氨酸氨基转移酶升高。处方信息载有肝毒性、胚胎与胎儿毒性，以及血清肌酐升高但不影响肾功能等警告与注意事项。
本次审评在 FDA 肿瘤学卓越中心的 Project Orbis 框架下进行，FDA 与澳大利亚治疗用品管理局、加拿大卫生部、以色列卫生部、瑞士医药管理局与英国药品与保健品监管局协作审评，欧洲药品管理局为观察员；审评还使用了申请人自愿提交的评估辅助材料。（来源：美国食品药品管理局）
FDA Approves Pimjetmi for Tenosynovial Giant Cell Tumor at 50 mg Once Daily
WASHINGTON — The U.S. Food and Drug Administration (FDA) on November 10 approved pimicotinib (Pimjetmi, EMD Serono, Inc.), a kinase inhibitor, for adults with symptomatic tenosynovial giant cell tumor (TGCT) for which surgical resection may cause worsening functional limitation or severe morbidity.
Efficacy was evaluated in MANEUVER (NCT05804045), a double-blind, randomized (2:1), placebo-controlled international trial in patients with a confirmed diagnosis of symptomatic TGCT with measurable disease, at least one lesion of at least 2 cm, and for whom surgical resection may cause dysfunction or serious complications. Patients were randomized, stratified by geographic region (China versus non-China), to pimicotinib 50 mg or placebo once daily.
The recommended dosage is 50 mg orally once daily, with or without food. Pimicotinib had received fast track and breakthrough therapy designations.
The most common adverse reactions (more than 20 percent) were pruritus, rash, face edema, periorbital edema, fatigue, nausea, headache, and peripheral edema. The most common laboratory abnormalities (more than 30 percent) were increased creatine phosphokinase, lactate dehydrogenase, aspartate aminotransferase, lipase, cholesterol, amylase, and alanine aminotransferase. The prescribing information includes warnings and precautions for hepatotoxicity, embryo-fetal toxicity, and increased serum creatinine without affecting renal function.
The review was conducted under Project Orbis, an initiative of the FDA Oncology Center of Excellence, in collaboration with the Australian Therapeutic Goods Administration, Health Canada, Israel's Ministry of Health, Switzerland's Swissmedic, and the United Kingdom's Medicines and Healthcare products Regulatory Agency, with the European Medicines Agency as an official observer. The review also used the Assessment Aid, a voluntary submission from the applicant. (Source: U.S. Food and Drug Administration)
来源：美国食品药品管理局
发布：GSM·美国（发布链接）
图片来源：图：美国食品药品监督管理局（FDA）位于马里兰州 Silver Spring 的 White Oak 园区总部大楼入口（FDA 官方图，公共领域，经 Wikimedia Commons）。 / Photo: The entrance to FDA headquarters at the White Oak campus in Silver Spring, Maryland (U.S. Food and Drug Administration, public domain, via Wikimedia Commons).
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