2016年12月19日，美国食品和药品监管局授权对Rubraca(rucaparib)加速批准治疗有某些类型卵巢癌妇女。Rubraca被批准为有晚期卵巢癌曽被两种或更多化疗治疗妇女和其肿瘤有一种特异性基因突变(有害的BRCA) 被一种FDA批准的协同诊断测试鉴定。
FDA的药品评价和研究中心中血液学和肿瘤办公室主任和FDA的卓越肿瘤中心代理主任Richard Pazdur，M.D.说：“今天的批准是我们正在见到开发靶向在在患者的基因中特异性突变引起的药物治疗癌症另一个实例，” “有这些基因异常对她们卵巢癌曽试用至少两种化疗治疗妇女现有一个另外的治疗选择。”
据美国癌症研究所估计在2016年22,280例妇女将被诊断有卵巢癌和被估计14,240将死于这个疾病。约15至20%有卵巢癌患者有一个BRCA基因突变。
BRCA基因是涉及修复被损伤的DNA和正常地工作以防止肿瘤发展。但是，这些基因的突变可导致某些癌症，包括卵巢癌。Rubraca是一种聚ADP-核糖聚合酶(PARP)抑制剂它阻断一种涉及修复受损伤DNA的酶。通过阻断这个酶。癌细胞内有被损伤BRCA基因DNA可能减低可能地被修复，导致细胞死亡和可能减慢或肿瘤生长停止。
今天，为与Rubraca使用FDA还批准FoundationFocus CDxBRCA 协同诊断，它是被监管局批准的第一个下-一代-基于测序(NGS)-协调诊断。NGS测试检测患者卵巢癌肿瘤组织中有害的BRCA基因突变的存在。如检测到一或更多突变，患者可能对用Rubraca治疗合格。
在两项，单-臂临床试验涉及106例有BRCA-突变的卵巢癌曽被用两或更多化疗方案治疗参加者中研究Rubraca的安全性和疗效。在96%被测试的试验参加者有可得到的肿瘤组织用FoundationFocus CDxBRCA协同诊断确证BRCA基因突变。本试验测量他们的肿瘤经历完全或部分皱缩参加者的百分率(总体反应率)。在本试验中44%接受Rubraca参加者他们的肿瘤经历完全或部分皱缩持续一个中位9.2个月。
Rubraca的常见不良反应包括恶心，疲乏，呕吐，贫血，腹痛，味觉障碍，便秘，食欲减退，腹泻，血小板减少和呼吸困难。Rubraca伴随有严重的风险例如骨髓问题(骨髓增生异常综合征)，一种典型血癌被称为急性髓性白血病和胎儿伤害。
监管局在其加速批准程序下批准Rubraca，该程序允许批准一个药物治疗一种严重的或危及生命疾病或情况根据临床数据显示药物有一种效应对一种替代性终点是合理地可能预测临床获益。承办单位正在有BRCA基因突变有晚期卵巢癌患者和在有卵巢癌其他类型患者继续研究该药。FDA还授予Rubraca申请突破性治疗指定和优先审评状态。Rubraca还接受孤儿药物指定，它提供鼓励例如税收减免，用户费用免除和专有权合格性有助于和鼓励意向治疗罕见疾病药物的开发。
Rubraca由总部在科罗拉多Boulder Clovis Oncology，有限公司制造。FoundationFocus CDxBRCA 协同诊断由麻省剑桥的Foundation Medicine有限公司上市。
FDA approved: Yes (First approved December 19th, 2016)
Brand name: Rubraca
Generic name: rucaparib
Dosage form: Tablets
Company: Clovis Oncology, Inc.
Treatment for: Ovarian Cancer
Rubraca (rucaparib) is a poly (ADP-ribose) polymerase (PARP) inhibitor indicated for the treatment of advanced mutant BRCA ovarian cancer.
CLOVIS ONCOLOGY ANNOUNCES FDA ACCELERATED APPROVAL OF RUBRACA™ (RUCAPARIB) FOR THE MONOTHERAPY TREATMENT OF ADVANCED OVARIAN CANCER IN WOMEN WITH DELETERIOUS GERMLINE OR SOMATIC BRCA MUTATIONS TREATED WITH TWO OR MORE
CHEMOTHERAPIES
•First and only PARP inhibitor in the U.S. indicated to treat advanced ovarian cancer patients who have been treated with two or more chemotherapies and who have deleterious germline or somatic BRCA mutations
•Rubraca received approval under the FDA’s accelerated approval program based on objective response rate and duration of response
•Most common Grade 3-4 adverse reaction was anemia; most common Grade 3-4 laboratory abnormality was decrease in hemoglobin
U.S. Food and Drug Administration (FDA) has approved Rubraca™ (rucaparib) tablets as monotherapy for the treatment of patients with deleterious BRCA mutation (germline and/or somatic) associated advanced ovarian cancer, who have been treated with two or more chemotherapies, and selected for therapy based on an FDA-approved companion diagnostic for Rubraca. Rubraca’s indication is approved under the FDA’s accelerated approval program, and is based on objective response rate and duration of response results from two multicenter, single-arm, open-label clinical trials, Study 10 and ARIEL2 Parts 1 and 2. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials. The ARIEL3 maintenance confirmatory study has completed enrollment and the ARIEL4 treatment |
