6月11日消息 - 环球医学据悉，美国食品和药物管理局于近日批准一种新的抗HER2疗法——Perjeta（帕妥珠单抗）用于治疗HER2-阳性的晚期（转移性）乳腺癌。

Perjeta旨在用于那些既往未接受过抗HER2疗法或化疗治疗的转移性乳腺癌患者；Perjeta与曲妥珠单抗（另一种抗HER2疗法）和多西他赛（一种化疗方法）联合用药。

HER2是一种参与正常细胞生长的蛋白质。在某些类型的癌细胞中，HER2的数量会增加（HER-2阳性），包括一些乳腺癌。在这些HER-2阳性乳腺癌中，HER2蛋白促成了癌细胞的生长和生存。

Perjeta是一种通过生物技术方法生产的人源化单克隆抗体。它通过静脉给药，并被认为作用于与曲妥珠单抗不同的靶点，从而使得HER-2阳性乳腺癌细胞的生长和生存进一步降低。

因为可能存在影响该药物长期供应的生产问题，FDA目前限制批准未受这些问题影响的药品。Perjeta的制造商基因泰克公司一直致力于设法及时解决这些生产问题。

“鉴于转移性乳腺癌需要额外的治疗，我们决定，在等待解决未来药品供应的相关生产问题时，批准该药并且不推迟该药的上市，”FDA药物评价与研究中心主任、医学博士 Janet Woodcock说。“基因泰克目前正在制定计划以缓解Perjeta潜在的短缺影响。”

乳腺癌是女性的第二大癌症相关死因。今年估计有226870名妇女将被诊断为乳腺癌，39510人将死于这种疾病。乳腺癌患者中，约20％的人HER2蛋白数量会增加。

“由于曲妥珠单抗最初是在十多年前获得的批准，所以持续不断的研究使得我们可以更好地了解HER2在乳腺癌中的作用，” FDA药物评价与研究中心血液学与肿瘤学制品办公室主任、医学博士、Richard Pazdur说。“这为曲妥珠单抗和Perjeta这两种靶向治疗药物联合多西他赛用于延缓乳腺癌的疾病进展提供了背景。”

一项涉及808例HER2阳性转移性乳腺癌患者的单一临床试验评价了Perjeta的安全性和有效性；这些患者在治疗前接受检查，以确定HER2蛋白是否增加。患者被随机分配接受Perjeta、曲妥珠单抗联合多西他赛，或是曲妥珠单抗、多西他赛联合安慰剂。

这项研究旨在是观察患者在癌症未进展的情况下存活的时长，即无进展生存期（PFS）。包含有Perjeta的联合治疗组的中位PFS为18.5个月，而包含有安慰剂的联合治疗组的中位PFS为12.4个月。

观察显示，接受Perjeta联合曲妥珠单抗和多西他赛治疗者最常见的副作用是腹泻、脱发、对抗感染的白细胞减少、恶心、乏力、皮疹和神经损伤（周围感觉神经病变）。

Perjeta获得了批准，但有一个黑框警告提醒患者和卫生保健专业人员注意潜在风险，包括死亡或对胎儿产生的严重影响。

该疗法是在FDA的优先审查程序下审评的，优先审查程序为可能带来治疗进展的药物提供6个月的加速审查。

Perjeta由罗氏集团的成员、总部位于旧金山的基因泰克公司销售。（环球医学）

原文：

FDA approves Perjeta for type of late-stage breast cancer

The U.S. Food and Drug Administration today approved Perjeta (pertuzumab), a new anti-HER2 therapy, to treat patients with HER2-positive late-stage (metastatic) breast cancer.

Intended for patients who have not received prior treatment for metastatic breast cancer with an anti-HER2 therapy or chemotherapy, Perjeta is combined with trastuzumab, another anti-HER2 therapy, and docetaxel, a type of chemotherapy.

HER2 is a protein involved in normal cell growth. It is found in increased amounts on some types of cancer cells (HER2-positive), including some breast cancers. In these HER2-positive breast cancers, the increased amount of the HER2 protein contributes to cancer cell growth and survival.

Perjeta is a humanized monoclonal antibody, manufactured through biotechnology methods. It is administered intravenously and is believed to work by targeting a different part of the HER-protein than trastuzumab, resulting in further reduction in growth and survival of HER2-positive breast cancer cells.

Because there are production issues that potentially could affect the long-term supply of the drug, FDA limited its approval today to drug product that has not been affected by those issues. Genentech, the manufacturer of Perjeta, has committed to take steps designed to resolve these production issues in a timely manner.

“Given the need for additional treatments for metastatic breast cancer, we made the decision to approve this drug today and not to delay its availability to patients pending resolution of the production issues relating to future supply,” said Janet Woodcock, M.D., director of FDA’s Center for Drug Evaluation and Research. “Genentech is currently developing a plan to mitigate the effect on patients of any potential shortage of Perjeta.”

Breast cancer is the second leading cause of cancer-related death among women. This year an estimated 226,870 women will be diagnosed with breast cancer, and 39,510 will die from the disease. About 20 percent of breast cancers have increased amounts of the HER2 protein.

“Since trastuzumab was first approved more than a decade ago, continued research has allowed us to better understand the role HER2 plays in breast cancer,” said Richard Pazdur, M.D., director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research. “This research provided the background to combine two targeted drugs – trastuzumab and Perjeta with docetaxel to slow disease progression in breast cancer.”

The safety and effectiveness of Perjeta were evaluated in a single clinical trial involving 808 patients with HER2-positive metastatic breast cancer who were tested prior to treatment to determine if the HER2 protein was increased. Patients were randomly assigned to receive Perjeta, trastuzumab and docetaxel or trastuzumab and docetaxel with a placebo.

The study was designed to measure the length of time a patient lived without the cancer progressing, progression-free survival (PFS). Those treated with the combination containing Perjeta had a median PFS of 18.5 months, while those treated with the combination containing placebo had a median PFS of 12.4.

The most common side effects observed in patients receiving Perjeta in combination with trastuzumab and docetaxel were diarrhea, hair loss, a decrease in infection-fighting white blood cells, nausea, fatigue, rash, and nerve damage (peripheral sensory neuropathy).

Perjeta is being approved with a Boxed Warning alerting patients and health care professionals to the potential risk of death or severe effects to a fetus. Pregnancy status must be verified prior to the start of Perjeta treatment.

The therapy was reviewed under the agency’s priority review program, which provides for an expedited six-month review of drugs that may offer major advances in treatment.

Perjeta is marketed by South San Francisco-based Genentech, a member of the Roche Group.
 

相关链接：http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm307549.htm 

版权声明：本文系环球医学独家稿件，版权为环球医学所有。欢迎转载，请务必注明出处（环球医学网），否则必将追究法律责任。