FDA官宣：百济神州泽布替尼审批意见！（附全文翻译）

FDA批准复发难治性套细胞淋巴瘤的治疗药，临床试验结果显示肿瘤缩小的有效率很高。

For Immediate Release:

November 14, 2019

Today, the U.S. Food and Drug Administration granted accelerated approval to Brukinsa (zanubrutinib) capsules for the treatment of adult patients with mantle cell lymphoma who have received at least one prior therapy.

今天，美国食品和药物管理局批准Brukinsa（Zanubrutinib）用于治疗至少接受过一次治疗的成人套细胞淋巴瘤患者。

“Mantle cell lymphoma usually responds well to initial treatment, but eventually returns or stops responding, and the cancer cells continue to grow. This is a life-threatening condition,” said Richard Pazdur, M.D., director of the FDA’s Oncology Center of Excellence and acting director of the Office of Oncologic Diseases in the FDA’s Center for Drug Evaluation and Research. “Clinical trials showed that 84% of patients saw tumor shrinkage with this therapy. For patients whose disease relapses or becomes refractory, secondary therapies may be successful in providing another remission, and today’s approval will provide patients with another treatment option.”

FDA肿瘤中心主任、FDA药物评价和研究中心肿瘤疾病办公室代理主任、医学博士Richard Pazdur表明：“套细胞淋巴瘤通常对最初的治疗反应良好，但最终会减缓或停止作用，癌细胞继续生长，这是一种危及生命的疾病。临床试验表明，84%的患者认为这种疗法会导致肿瘤缩小。对于疾病复发或变得难治的患者，二次治疗可能会成功地提供另一种缓解，今天的批准将为患者提供另一种治疗选择。”

Mantle cell lymphoma is a type of non-Hodgkin’s lymphoma representing 3-10% of all non-Hodgkin’s lymphomas in the United States. By the time it is diagnosed, mantle cell lymphoma has usually spread to the lymph nodes, bone marrow and other organs. In relapsed lymphoma, the disease reappears or grows again after a period of remission, while in refractory lymphoma, the disease does not respond to treatment or responds only briefly.

套细胞淋巴瘤是一种非霍奇金淋巴瘤，占美国非霍奇金淋巴瘤的3-10%。到确诊时，套细胞淋巴瘤通常已经扩散到淋巴结、骨髓和其他器官。在复发性淋巴瘤中，该病在一段时间的缓解后再次出现或生长，而在难治性淋巴瘤中，该病对治疗无反应或仅短暂反应。

A single-arm clinical trial of Brukinsa included 86 patients with mantle cell lymphoma who had received at least one prior treatment. The trial measured how many patients experienced complete or partial shrinkage of their tumors after treatment (overall response rate). In the trial, 84% of patients had tumor shrinkage with a median duration of response (time between the initial response to therapy and subsequent disease progression or relapse) of 19.5 months. This trial was supported by an additional single-arm trial that included 32 patients, in which 84% of patients had tumor shrinkage with a median duration of response of 18.5 months.

Brukinsa的随访临床试验包括86例经至少一次治疗的套细胞淋巴瘤患者。该试验测量了多少患者在治疗后肿瘤完全或部分缩小（总体响应率）。在试验中，84%的患者肿瘤缩小，反应持续时间中位数（从治疗的最初反应到随后的疾病进展或复发之间的时间）为19.5个月。这项试验得到了另一项包括32名患者的随访试验的支持，其中84%的患者肿瘤缩小，缓解时间中位数为18.5个月。

Common side effects for patients taking Brukinsa were decreased neutrophil count (white blood cells that fight against infection), decreased platelet count (a component of blood whose function is to react to bleeding from blood vessel injury by clumping, initiating a blood clot), upper respiratory tract infection, decreased white blood cell count, decreased hemoglobin (oxygen-carrying protein in red blood cells), rash, bruising, diarrhea and cough. During treatment, patients should be monitored for hemorrhage (bleeding), signs and symptoms of infection, cytopenias (decreased complete blood counts) and cardiac arrhythmias (irregular, rapid heart rate and abnormality in beating of the heart). Patients are advised to use sun protection if taking this therapy because there is a risk of other malignancies occurring including skin cancers. The FDA advises health care professionals to tell females of reproductive age and males with a female partner of reproductive potential to use effective contraception during treatment with Brukinsa. Women who are pregnant or breastfeeding should not take Brukinsa because it may cause harm to a developing fetus or newborn baby.

服用Brukinsa的患者的常见副作用是中性粒细胞数（对抗感染的白细胞）、血小板数（血液的一种成分，其功能是对血管损伤引起的出血作出反应，形成血栓）、上呼吸道感染、白细胞数下降，血红蛋白（红细胞中的含氧蛋白）、皮疹、瘀伤、腹泻和咳嗽减少。在治疗过程中，应监测患者的出血、感染症状和体征、细胞减少（全血减少）和心律失常（心率不规则、快速和心跳异常）。如果患者接受这种疗法，建议使用防晒霜，因为有可能发生其他恶性肿瘤，包括皮肤癌。FDA建议卫生保健专业人员告诉育龄女性和有生殖潜能的女性伴侣的男性在治疗Brukinsa期间使用有效的避孕措施。孕妇或哺乳期妇女不应服用Brukinsa，因为它可能对发育中的胎儿或新生儿造成伤害。

Brukinsa was granted Accelerated Approval, which enables the FDA to approve drugs for serious conditions to fill an unmet medical need based on a result that is reasonably likely to predict a clinical benefit to patients. Further clinical trials may be required to verify and describe Brukinsa’s clinical benefit.

Brukinsa获得了加速批准，这使得FDA能够批准重要情况下的药物，以满足未满足的医疗需求，其结果有可能合理预测患者的临床益处。可能需要进一步的临床试验来验证和描述Brukinsa的临床益处。

The FDA granted this application Breakthrough Therapy designation, which expedites the development and review of drugs that are intended to treat a serious condition, and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapies. Brukinsa also received Orphan Drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases. The FDA granted approval of Brukinsa to BeiGene USA Inc.

FDA批准了这一突破性的治疗方案，加速了治疗严重疾病药物的开发和审查，初步临床证据表明，该药物可能比现有疗法有实质性的改善。Brukinsa还获得了罕见病用药指定，这为帮助和鼓励开发治疗罕见疾病的药物提供了奖励。FDA批准了百济神州美国分公司生产Brukinsa。

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是美国卫生和公共服务部的一个机构，通过确保人用、兽药、疫苗和其他生物制品以及医疗器械的安全性、有效性和安全性来保护公众健康。该机构还负责美国食品供应、化妆品、膳食补充剂、释放电子辐射的产品的安全和保障，以及管制烟草产品。

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