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FDA 批准了首款PI3K抑制剂用于治疗乳腺癌

FDA approves first PI3K inhibitor for breast cancer
FDA 批准了首款PI3K抑制剂用于治疗乳腺癌
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2019-07-04 13:29
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FDA 批准了首款PI3K抑制剂用于治疗乳腺癌

For Immediate Release: May 24, 2019

立即发布:2019年5月24日

Today, the U.S. Food and Drug Administration approved Piqray (alpelisib) tablets, to be used in combination with the FDA-approved endocrine therapy fulvestrant, to treat postmenopausal women, and men, with hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative, PIK3CA-mutated, advanced or metastatic breast cancer (as detected by an FDA-approved test) following progression on or after an endocrine-based regimen.

今天,美国食药监局批准了Piqray(alpelisib) 与另一款食药监局批准的内分泌治疗药物氟维司群联合用于治疗,经FDA认证的检查后发现的激素受体(HR)-阳性,人类表皮生长因子2(HER2)-阴性,具PIK3CA-突变,在内分泌治疗期间或之后出现疾病进展的绝经后女性及男性晚期或转移性乳腺癌患者。

The FDA also approved the companion diagnostic test, therascreen PIK3CA RGQ PCR Kit, to detect the PIK3CA mutation in a tissue and/or a liquid biopsy. Patients who are negative by the therascreen test using the liquid biopsy should undergo tumor biopsy for PIK3CA mutation testing.

therascreen PIK3CA RGQ PCR Kit伴随诊断检查也通过了食药监局的批准, 用来在组织活检或者血液样本中检测PIK3CA突变。那些通过血液样本做的therascreen检测结果为阴性的患者,需要再通过组织活检做一次PIK3CA突变测试。

“Piqray is the first PI3K inhibitor to demonstrate a clinically meaningful benefit in treating patients with this type of breast cancer. The ability to target treatment to a patient’s specific genetic mutation or biomarker is becoming increasingly common in cancer treatment, and companion diagnostic tests assist oncologists in selecting patients who may benefit from these targeted treatments,” said Richard Pazdur, M.D., director of the FDA’s Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research. “For this approval, we employed some of our newer regulatory tools to streamline reviews without compromising the quality of our assessment. This drug is the first novel drug approved under the Real-Time Oncology Review pilot program. We also used the updated Assessment Aid, a multidisciplinary review template that helps focus our written review on critical thinking and consistency and reduces time spent on administrative tasks.”

Piqray 是首款PI3K抑制剂为用于治疗此种类型(HR+,HER2-,PIK3CA 突变)的乳腺癌患者获得临床获益提供了证据。FDA卓越肿瘤中心主任,FDA血液与肿瘤产品药物评定与研究中心执行主任, Richard Pazdur 博士说:“针对患者的某个突变基因或者生物标记物来实施靶向治疗正越来越普及,同时,伴随诊断能够协助肿瘤医生挑选出更能够从这些靶向治疗中获益的患者。 为了这项审批,我们启用了一些新的管理工具去简化流程但不降低评估标准。Piqray 是第一个通过Real-Time 肿瘤评估试点项目(RTOR)批准的新药。我们也使用了新的评估助手(AAID),那是一款多学科评估模板,从而帮助我们关注书面报告的判断性思维和药物的一致性评价上,并且降低了花费在行政任务上的时间。”

Metastatic breast cancer is breast cancer that has spread beyond the breast to other organs in the body (most often the bones, lungs, liver or brain). When breast cancer is hormone-receptor positive, patients may be treated with anti-hormonal treatment (also called endocrine therapy), alone or in combination with other medicines, or chemotherapy.

转移性乳腺癌是已经转移到乳腺以外身体其他部位的乳腺癌(通常会转移到骨,肺,肝或者脑)。乳腺癌激素受体阳性的患者可以接受单独的抗激素治疗(也被称为内分泌治疗),或者联合其他药物治疗,或者化疗。

The efficacy of Piqray was studied in the SOLAR-1 trial, a randomized trial of 572 postmenopausal women and men with HR-positive, HER2-negative, advanced or metastatic breast cancer whose cancer had progressed while on or after receiving an aromatase inhibitor. Results from the trial showed the addition of Piqray to fulvestrant significantly prolonged progression- free survival (median of 11 months vs. 5.7 months) in patients whose tumors had a PIK3CA mutation.

Piqray 被用于一项随机试验,入组了激素受体阳性,HER-2阴性,晚期或者转移性乳腺癌,正在接受芳香化酶抑制剂或往期使用过芳香化酶抑制剂后疾病进展的572名绝经后女性和男性。结果显示,Piqray相比于氟维司群显著延长了PIK3CA突变患者的无进展生存期(中位数:11个月和5.7个月)。

Common side effects of Piqray are high blood sugar levels, increase in creatinine, diarrhea, rash, decrease in lymphocyte count in the blood, elevated liver enzymes, nausea, fatigue, low red blood cell count, increase in lipase (enzymes released by the pancreas), decreased appetite, stomatitis, vomiting, weight loss, low calcium levels, aPTT prolonged (blood clotting taking longer to occur than it should), and hair loss.

Piqray普遍的副作用有高血糖,肌酐升高,腹泻,皮疹,淋巴细胞计数降低,肝酶升高,恶心,疲劳,低血红细胞,脂肪酶升高(一种胰腺释放的酶),食欲降低,口腔炎,呕吐,消瘦,低血钙,凝血时间延长(血液凝固时间比正常所需时间要长),掉发。

Health care professionals are advised to monitor patients taking Piqray for severe hypersensitivity reactions (intolerance). Patients are warned of potentially severe skin reactions (rashes that may result in peeling and blistering of skin or mucous membranes like the lips and gums). Health care professionals are advised not to initiate treatment in patients with a history of severe skin reactions such as Stevens-Johnson Syndrome, erythema multiforme, or toxic epidermal necrolysis. Patients on Piqray have reported severe hyperglycemia (high blood sugar), and the safety of Piqray in patients with Type 1 or uncontrolled Type 2 diabetes has not been established. Before initiating treatment with Piqray, health care professionals are advised to check fasting glucose and HbA1c, and to optimize glycemic control. Patients should be monitored for pneumonitis/interstitial lung disease (inflammation of lung tissue) and diarrhea during treatment. Piqray must be dispensed with a patient Medication Guide that describes important information about the drug’s uses and risks.

建议主治医生需要管理病人在服用Piqray期间发生的严重的过敏反应(不耐受)。告诫患者潜在的严重皮肤反应(皮疹可能造成皮肤或黏膜的蜕皮和起泡,比如嘴唇和牙龈)。建议主治医生不要将Piqray用于患有严重皮肤疾病的患者如StevensJohnson综合征,多形性红斑,中毒性表皮坏死溶解症的患者的初始治疗。服用Piqray的病人被报道过有严重的高糖血症(高血糖)并且在I型或未被控制的II型糖尿病的患者中使用Piqray的安全性剂量还未被测试。在使用Piqray进行首次治疗前,建议主治医生先行检测患者的空腹血糖和HbA1c(糖化血红蛋白)并且将患者的血糖水平控制在最佳状态。在治疗过程中患者需要管控好间质性肺炎(肺部组织的炎症)和腹泻。Piqray 必须由用药向导(药剂师或主治医生)向患者讲述关于药品的使用方法和副作用的重要信息后分发给患者。

Piqray is the first new drug application (NDA) for a new molecular entity approved under the Real-Time Oncology Review (RTOR) pilot program, which permits the FDA to begin analyzing key efficacy and safety datasets prior to the official submission of an application, allowing the review team to begin their review and communicate with the applicant earlier. Piqray also used the updated Assessment Aid (AAid), a multidisciplinary review template intended to focus the FDA’s written review on critical thinking and consistency and reduce time spent on administrative tasks. With these two pilot programs, today’s approval of Piqray comes approximately three months ahead of the Prescription Drug User Fee Act (PDUFA) VI deadline of August 18, 2019.

Piqray是第一个新的药物申请(NDA)的新分子实体批准的实时肿瘤学审查(RTOR)试点计划,此计划允许FDA在正式提交申请前开始分析关键疗效和安全数据集,审查小组开始审查并提前与申请人沟通。Piqray还使用了最新的评估辅助(AAID),这是一个多学科审查模板,旨在将FDA的审查集中在对书面报告的批判性思维和药物的一致性评价上,并减少花在行政任务上的时间。有了这两个试点项目,今天批准piqray的时间比处方药使用法案(PDUFA)第六次截止日期(2019年8月18日)提前了大约三个月。

The FDA granted this application Priority Review designation. The FDA granted approval of Piqray to Novartis. The FDA granted approval of the therascreen PIK3CA RGQ PCR Kit to QIAGEN Manchester, Ltd.

FDA批准了这项申请优先评估的指定。FDA批准了诺华的Piqray.FDA批准了 曼彻斯特有限公司的伴随诊断 therascreen PIK3CA RGQ PCR Kit

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.

FDA是美国卫生和人类服务机构,通过确保人类和兽药,疫苗和其他工人类使用的生物产品以及医疗谁被的安全性,有效性来维护公共卫生。这一机构也负责美国的食品供应,化妆品,膳食补充剂,电离辐射产品和常规的烟草产品的安全和安保。

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