FDA批准对慢性移植物抗宿主病治疗药物-Imbruvicad-2017.08.02

FDA approves treatment for chronic graft versus host disease


For Immediate Release
August 2, 2017


The U.S. Food and Drug Administration today expanded the approval of Imbruvica (ibrutinib) for the treatment of adult patients with chronic graft versus host disease (cGVHD) after failure of one or more treatments. This is the first FDA-approved therapy for the treatment of cGVHD.


美国食品和药物管理局今天详述了Imbruvica(ibruvicb)的批准，用于治疗慢性移植物抗宿主病(cGVHD)的**患者，在一次或多个治疗失败后。这是第一个fda批准的治疗cGVHD的疗法。


cGVHD is a life-threatening condition that can occur in patients after they receive a stem cell transplant from blood or bone marrow, called hematopoietic stem cell transplantation (HSCT), to treat certain blood or bone marrow cancers. cGVHD occurs when cells from the stem cell transplant attack healthy cells in patient's tissues. Symptoms of cGVHD can occur in the skin, eyes, mouth, gut, liver and lungs. The condition is estimated to occur in 30-70 percent of all patients who receive HSCT.


为治疗某些血液或骨髓癌症，病人在接受血液或骨髓的干细胞移植，也叫做造血干细胞移植后，cGVHD会发生在病人身上，这对他们来说这是一种生命的威胁。当来自于干细胞移植的细胞攻病人机体组织中健康的细胞时，cGVHD就会发生。cGVHD的症状可出现在皮肤、眼睛、嘴巴、肠、肝脏和肺。据估计，在接受HSCT治疗的所有患者中，有30%-70%的人会出现这种情况。


“Patients with cGVHD who do not respond to other forms of therapy-typically corticosteroids to suppress their immune system-now have a treatment option specifically indicated to treat their condition,” 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. “This approval highlights how a known treatment for cancer is finding a new use in treating a serious and life-threatening condition that may occur in patients with blood cancer who receive a stem cell transplant.”


Richard Pazdur说:“患有cGVHD的病人，他们不会对其他形式的治疗-典型的皮质激素-来抑制他们的免疫系统-现在有了一个特殊的治疗方案来治疗他们的病情。”他是FDA肿瘤研究中心的主任，同时也是FDA药物评估和研究中心的血液学和肿瘤产品办公室的代理主任。“这一批准突出表明，一种已知的癌症治疗方法正在寻找一种新的治疗方法，用于治疗在接受干细胞移植的患者中可能出现的严重和危及生命的疾病。

The efficacy and safety of Imbruvica for the treatment of cGVHD were studied in a single-arm trial of 42 patients with cGVHD whose symptoms persisted despite standard treatment with corticosteroids. Most patients’ symptoms included mouth ulcers and skin rashes, and more than 50 percent of patients had two or more organs affected by cGVHD. In the trial, 67 percent of patients experienced improvements in their cGVHD symptoms. In 48 percent of patients in the trial, the improvement of symptoms lasted for up to five months or longer.摘要对cGVHD的治疗效果和安全性进行了研究，对42名cGVHD患者进行了单臂试验，尽管采用了皮质类固醇激素治疗，但其症状仍然存在。大多数患者的症状包括口腔溃疡和皮疹，超过50%的患者有两个或更多的器官受到了cGVHD的影响。在试验中，67%的患者经历了cGVHD症状的改善。在48%的患者中，症状的改善持续了5个月甚至更长。

Common side effects of Imbruvica in patients with cGVHD include fatigue, bruising, diarrhea, low levels of blood platelets (thrombocytopenia), muscle spasms, swelling and sores in the mouth (stomatitis), nausea, severe bleeding (hemorrhage), low levels of red blood cells (anemia) and lung infection (pneumonia).

对cGVHD患者的常见副作用包括:疲劳、瘀伤、腹泻、低水平血小板(血小板减少)、肌肉痉挛、肿胀和口腔溃疡(口腔炎)、恶心、严重出血(出血)、低水平的红细胞(贫血)和肺部感染(肺炎)。

Serious side effects of Imbruvica include severe bleeding (hemorrhage), infecti**, low levels of blood cells (cytopenias), irregular heartbeat (atrial fibrillation), high blood pressure (hypertension), new cancers (second primary malignancies) and metabolic abnormalities (tumor lysis syndrome). Women who are pregnant or breastfeeding should not take Imbruvica because it may cause harm to a developing fetus or a newborn baby.

Imbruvica的严重副作用包括严重出血(出血)、感染、低血细胞(细胞内)、不规则心跳(心房纤维性颤动)、高血压(高血压)、新癌症(第二次原发性恶性肿瘤)和代谢异常(肿瘤溶解症)。怀孕或哺乳的妇女不应服用Imbruvica，因为它可能对发育中的胎儿或新生儿造成伤害。

Imbruvica, a kinase inhibitor, was previously approved for certain indicati** in treating chronic lymphocytic leukemia, Waldenström’s macroglobulinemia and marginal zone lymphoma, as well as under accelerated approval status for mantle cell lymphoma.

Imbruvica，一种激酶抑制剂，以前曾被批准用于治疗慢性淋巴细胞白血病，waldenstrm的大细胞瘤和边缘区域淋巴瘤，以及在地幔细胞淋巴瘤的加速批准状态下。

The FDA granted this application Priority Review and Breakthrough Therapydesignati**. Imbruvica also received Orphan Drug designation for this indication, which provides incentives to assist and encourage the development of drugs for rare diseases.

美国食品和药物管理局批准了这一申请的优先审查和突破性的治疗。Imbruvica还获得了“孤儿药”的称号，这一指示提供了鼓励和鼓励治疗罕见疾病的药物。

The FDA granted the approval of Imbruvica to Pharmacyclics LLC.

FDA授权批准了Imbruvica于Pharmacyclics LLC。

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 resp**ible 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是美国卫生和公众服务部的一个机构，它通过确保人类和兽药、疫苗和其他生物制品的安全性、有效性和安全性来保护公众健康，并确保人类使用和医疗设备的安全。该机构还负责国家的食品供应、化妆品、膳食补充剂、电子辐射产品以及烟草产品的安全。