On October 11, 2023, the Food and Drug Administration approved encorafenib (Braftovi, Array BioPharma Inc., a wholly owned subsidiary of Pfizer) with binimetinib (Mektovi, Array BioPharma Inc.) for adult patients with metastatic non-small cell lung cancer (NSCLC) with a BRAF V600E mutation, as detected by an FDA-approved test.FDA also approved the FoundationOne CDx (tissue) and FoundationOne Liquid CDx (plasma) as companion diagnostics for encorafenib with binimetinib.

2023年10月11日，美国食品和药物管理局批准encorafenib（Braftovi，Array BioPharma Inc.，辉瑞的合资子公司）与binimetinib（Mektovi，Array BioPharma Inc.）用于成人转移性非小细胞肺癌患者（NSCLC）具有BRAF V600E突变，如通过FDA批准的测试所检测的。FDA还批准FoundationOne CDx（组织）和FoundationOne液体CDx（血浆）作为encorafenib与binimetinib的伴随诊断。

If no mutation is detected in a plasma specimen, the tumor tissue should be tested.Efficacy was evaluated in 98 patients with metastatic NSCLC with BRAF V600E mutation enrolled in PHAROS (NCT03915951), an open-label, multicenter, single-arm study. Prior BRAF or MEK inhibitors was not allowed. Patients received encorafenib and binimetinib until disease progression or unacceptable toxicity.The major efficacy outcome measures were objective response rate (ORR) per RECIST v1.1 and duration of response (DoR), as assessed by an independent review committee.

如果在血浆样本中未检测到突变，则应测试肿瘤组织。在98名患有BRAF V600E突变的转移性NSCLC患者中评估了疗效，该患者参加了PHAROS（NCT03915951），这是一项开放标签，多中心，单臂研究。以前的BRAF或MEK抑制剂是不允许的。患者接受encorafenib和binimetinib直至疾病进展或不可接受的毒性。主要疗效结果指标是独立审查委员会评估的每个RECIST v1.1的客观缓解率（ORR）和缓解持续时间（DoR）。

Among 59 treatment-naïve patients, ORR was 75% (95% CI: 62, 85); median DoR was not estimable (NE) (95% CI: 23.1, NE). Among 39 previously treated patients, ORR was 46% (95% CI: 30, 63) with a median DoR of 16.7 months (95% CI: 7.4, NE).The most common adverse reactions (≥25%) were fatigue, nausea, diarrhea, musculoskeletal pain, vomiting, abdominal pain, visual impairment, constipation, dyspnea, rash, and cough.The recommended doses for BRAF V600E mutation-positive NSCLC are encorafenib 450 mg orally once daily and binimetinib 45 mg orally twice daily.This review used the Assessment Aid, a voluntary submission from the applicant to facilitate the FDA’s assessment.This application was granted orphan drug designation.

在59名未接受过治疗的患者中，ORR为75%（95%CI:62,85）；中位DoR不可估计（NE）（95%CI:23.1，NE）。在之前接受治疗的39例患者中，ORR为46%（95%CI:30,63），中位DoR为16.7个月（95%CI:7.4，NE）。最常见的不良反应（≥25%）为疲劳，恶心腹泻，肌肉骨骼疼痛，呕吐，腹痛，视力障碍，便秘，呼吸困难，皮疹和咳嗽。BRAF V600E突变阳性NSCLC的推荐剂量为每日口服一次450毫克encorafenib和每日两次口服45毫克binimetinib。这次审查使用了评估援助，这是申请人自愿提交的，以促进FDA的评估。该申请被授予孤儿药名称。

FDA expedited programs are described in t.

FDA加速计划在t中描述。