美国食品药品管理局11月10日批准匹米替尼用于症状性腱鞘巨细胞瘤：MANEUVER试验第25周总缓解率56%对3.2%
美国食品药品管理局11月10日批准匹米替尼，商品名Pimjetmi，由EMD Serono公司销售，是一种激酶抑制剂，用于手术切除可能导致功能受限加重或严重并发症的成人症状性腱鞘巨细胞瘤。该局当日批准页列明上述适应症与企业名。
疗效在MANEUVER试验中评估，试验编号NCT05804045，为双盲、随机2比1、安慰剂对照的国际试验，入组经确认的症状性腱鞘巨细胞瘤患者，病灶可测量且至少一处病灶最小尺寸为2厘米。患者每日一次服用匹米替尼50毫克或安慰剂，随机按地理区域即中国与非中国分层。
主要疗效指标为第25周按RECIST v1.1标准、经盲态独立中心评审的总缓解率。匹米替尼组总缓解率为56%，安慰剂组为3.2%。匹米替尼组中位缓解持续时间未达到，32名缓解者即91%的缓解持续时间达6个月以上，27名即77%达12个月以上。该局称，受累关节活动度、最重疼痛与身体功能等预设患者报告与功能终点在第25周亦有统计学显著改善。
该局列明，最常见的不良反应即发生率高于20%的包括瘙痒、皮疹、面部水肿、眶周水肿、疲劳、恶心、头痛与外周水肿。最常见的实验室异常即高于30%的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。处方信息含肝毒性、胚胎胎儿毒性与血清肌酐升高但不影响肾功能的警告。推荐剂量为每日一次口服50毫克，可与食物同服或空腹服用。
本次审评在食品药品管理局肿瘤卓越中心的Orbis项目下进行，与澳大利亚、加拿大、以色列、瑞士与英国监管机构协作，欧洲药品管理局为观察员。该药获快速通道与突破性疗法认定。本稿只报道该局批准页所载事实与资源信息，不作个体诊疗建议，完整处方信息将刊于Drugs@FDA。
FDA Approves Pimicotinib on November 10 for Symptomatic Tenosynovial Giant Cell Tumor: Overall Response Rate 56 Percent Versus 3.2 Percent at Week 25 in MANEUVER
On November 10, 2026, the Food and Drug Administration approved pimicotinib (Pimjetmi, EMD Serono, Inc.), a kinase inhibitor, for adults with symptomatic tenosynovial giant cell tumor for which surgical resection may cause worsening functional limitation or severe morbidity, according to the agency approval page posted that day.
Efficacy was evaluated in MANEUVER (NCT05804045), a double-blind, randomized 2 to 1, placebo-controlled international trial in patients with confirmed symptomatic disease with measurable disease and at least one lesion of at least 2 cm. Patients received pimicotinib 50 mg or placebo once daily, with randomization stratified by geographic region, China versus non-China.
The major efficacy outcome was overall response rate by blinded independent central review per RECIST v1.1 at Week 25. The rate was 56 percent in the pimicotinib arm and 3.2 percent in the placebo arm. Median duration of response was not reached in the pimicotinib arm; 32 responders, or 91 percent, had a duration of at least 6 months and 27, or 77 percent, had at least 12 months. The agency said statistically significant improvements were also seen at Week 25 in active range of motion, worst pain and physical functioning endpoints.
The agency listed the most common adverse reactions, above 20 percent, as pruritus, rash, face edema, periorbital edema, fatigue, nausea, headache and peripheral edema. The most common laboratory abnormalities, above 30 percent, included increased creatine phosphokinase, lactate dehydrogenase, aspartate aminotransferase, lipase, cholesterol, amylase and alanine aminotransferase. The prescribing information includes warnings for hepatotoxicity, embryo-fetal toxicity and increased serum creatinine without affecting renal function. The recommended dosage is 50 mg orally once daily with or without food.
The review was conducted under Project Orbis of the FDA Oncology Center of Excellence, in collaboration with regulators in Australia, Canada, Israel, Switzerland and the United Kingdom, with the European Medicines Agency as an observer. The drug received fast track and breakthrough therapy designations. This report covers only the facts and resource information on the agency approval page and makes no individual treatment recommendation. Full prescribing information will be posted on Drugs@FDA.
来源链接： https://www.fda.gov/drugs/cancer-approvals/fda-approves-pimicotinib-symptomatic-tenosynovial-giant-cell-tumor
发布：GSM·美国（发布链接）
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