FDA批準(zhǔn)復(fù)發(fā)難治性套細(xì)胞淋巴瘤的治療藥，臨床試驗(yàn)結(jié)果顯示腫瘤縮小的有效率很高。

       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，美國(guó)食品和藥物管理局批準(zhǔn)Brukinsa（Zanubrutinib）用于治療至少接受過一次治療的成人套細(xì)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腫瘤中心主任、FDA藥物評(píng)價(jià)和研究中心腫瘤疾病辦公室代理主任、醫(yī)學(xué)博士Richard Pazdur表明：“套細(xì)胞淋巴瘤通常對(duì)最初的治療反應(yīng)良好，但最終會(huì)減緩或停止作用，癌細(xì)胞繼續(xù)生長(zhǎng)，這是一種危及生命的疾病。臨床試驗(yàn)表明，84%的患者認(rèn)為這種療法會(huì)導(dǎo)致腫瘤縮小。對(duì)于疾病復(fù)發(fā)或變得難治的患者，二次治療可能會(huì)成功地提供另一種緩解，今天的批準(zhǔn)將為患者提供另一種治療選擇。”

       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.

       套細(xì)胞淋巴瘤是一種非霍奇金淋巴瘤，占美國(guó)非霍奇金淋巴瘤的3-10%。到確診時(shí)，套細(xì)胞淋巴瘤通常已經(jīng)擴(kuò)散到淋巴結(jié)、骨髓和其他器官。在復(fù)發(fā)性淋巴瘤中，該病在一段時(shí)間的緩解后再次出現(xiàn)或生長(zhǎng)，而在難治性淋巴瘤中，該病對(duì)治療無反應(yīng)或僅短暫反應(yīng)。

       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的隨訪臨床試驗(yàn)包括86例經(jīng)至少一次治療的套細(xì)胞淋巴瘤患者。該試驗(yàn)測(cè)量了多少患者在治療后腫瘤完全或部分縮小（總體響應(yīng)率）。在試驗(yàn)中，84%的患者腫瘤縮小，反應(yīng)持續(xù)時(shí)間中位數(shù)（從治療的最初反應(yīng)到隨后的疾病進(jìn)展或復(fù)發(fā)之間的時(shí)間）為19.5個(gè)月。這項(xiàng)試驗(yàn)得到了另一項(xiàng)包括32名患者的隨訪試驗(yàn)的支持，其中84%的患者腫瘤縮小，緩解時(shí)間中位數(shù)為18.5個(gè)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的患者的常見副作用是中性粒細(xì)胞數(shù)（對(duì)抗感染的白細(xì)胞）、血小板數(shù)（血液的一種成分，其功能是對(duì)血管損傷引起的出血作出反應(yīng)，形成血栓）、上呼吸道感染、白細(xì)胞數(shù)下降，血紅蛋白（紅細(xì)胞中的含氧蛋白）、皮疹、瘀傷、腹瀉和咳嗽減少。在治療過程中，應(yīng)監(jiān)測(cè)患者的出血、感染癥狀和體征、細(xì)胞減少（全血減少）和心律失常（心率不規(guī)則、快速和心跳異常）。如果患者接受這種療法，建議使用防曬霜，因?yàn)橛锌赡馨l(fā)生其他惡性腫瘤，包括皮膚癌。FDA建議衛(wèi)生保健專業(yè)人員告訴育齡女性和有生殖潛能的女性伴侶的男性在治療Brukinsa期間使用有效的避孕措施。孕婦或哺乳期婦女不應(yīng)服用Brukinsa，因?yàn)樗赡軐?duì)發(fā)育中的胎兒或新生兒造成傷害。

       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獲得了加速批準(zhǔn)，這使得FDA能夠批準(zhǔn)重要情況下的藥物，以滿足未滿足的醫(yī)療需求，其結(jié)果有可能合理預(yù)測(cè)患者的臨床益處。可能需要進(jìn)一步的臨床試驗(yàn)來驗(yàn)證和描述Brukinsa的臨床益處。

       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批準(zhǔn)了這一突破性的治療方案，加速了治療嚴(yán)重疾病藥物的開發(fā)和審查，初步臨床證據(jù)表明，該藥物可能比現(xiàn)有療法有實(shí)質(zhì)性的改善。Brukinsa還獲得了罕見病用藥指定，這為幫助和鼓勵(lì)開發(fā)治療罕見疾病的藥物提供了獎(jiǎng)勵(lì)。FDA批準(zhǔn)了百濟(jì)神州美國(guó)分公司生產(chǎn)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是美國(guó)衛(wèi)生和公共服務(wù)部的一個(gè)機(jī)構(gòu)，通過確保人用、獸藥、**和其他生物制品以及醫(yī)療器械的安全性、有效性和安全性來保護(hù)公眾健康。該機(jī)構(gòu)還負(fù)責(zé)美國(guó)食品供應(yīng)、化妝品、膳食補(bǔ)充劑、釋放電子輻射的產(chǎn)品的安全和保障，以及管制煙草產(chǎn)品。