首页 > 资讯 > FDA批准Imbruvica用于罕见血癌治疗

出自识林

2013-11-18 FDA

FDA批准第二个突破性疗法认定药品Imbruvica用于罕见血癌治疗
2013年11月13日

美国食品药品管理局（FDA）于日前批准Imbruvica (Ibrutinib)用于套细胞淋巴瘤(MCL)患者的治疗，该疾病属于一种罕见的侵润性血癌。

MCL是一种罕见的非霍奇金氏淋巴瘤，在美国所有非霍奇金氏淋巴瘤病例中大约占到6%。确诊为MCL时，通常已扩散至淋巴结、骨髓和其它器官。

Imbruvica拟用于之前已接受过至少一种治疗的MCL患者, Imbruvica通过抑制癌症增生与扩散所需要的酶而起作用。Imbruvica是第三种获批用于治疗MCL的药品。此前，万珂 (Velcade)和来那度胺(Revlimid)分别于2006年和2013年获批用于治疗该病。

“Imbruvica的获批显示了FDA致力于让罕见病患者获得治疗药品”，FDA药物审评与研究中心血液学和肿瘤学产品办公室主任、医学博士Richard Pazdur表示。“FDA与制药公司协作加快药品研发、审评和批准，体现了突破性治疗药物认定计划的承诺。”

Imbruvica是第二个以突破性治疗药物认定【FDA突破性治疗路径 识林资讯】形式获得FDA批准的药品。2012年通过的《FDA安全与创新法案》，使得FDA可在下述情况下认定一种药品为突破性治疗药品：如果初步临床证据显示，与现有可用治疗相比，一种药品可以给严重或危及生命的疾病患者提供一种确实改善，药品申请人可向FDA提出突破性治疗药品认定请求。

FDA按照加速审评计划批准Imbruvica，该计划准许FDA根据药品对可合理预测患者临床受益替代终点有效的临床试验数据，批准药品用于治疗严重病症。该计划可以使患者更早获得有前景的新药，而公司需要开展确证性临床试验。由于该药品显示出在治疗严重病症时安全性或有效性方面的协助改善的潜力，并计划用于治疗罕见疾病，FDA也给予Imbruvica优先审批和罕见病治疗用药认定。

Imbruvica用于MCL治疗的加速审批，是基于一项有111名受试者参与的临床研究，受试者每天接受Imbruvica治疗，直至疾病进展或副作用变得无法忍受为止。结果显示，约66%的受试者在治疗后癌症缩小或消失（总响应率）。生存率或与疾病相关症状情况尚未建立。

使用Imbruvica治疗的受试者中，最为常见的副作用是血液中血小板水平降低(血小板减少症)、腹泻、抗感染白细胞数量降低(中性粒细胞减少症)、贫血、疲劳、肌肉骨骼痛、肿胀(水肿)、上呼吸道感染、恶心、瘀伤、呼吸局促(呼吸困难)、便秘、皮疹、腹痛、呕吐和食欲不振。其它临床上明显的副作用包括出血、感染、肾病和出现其它类型癌症。

Imbruvica由总部位于加州Sunnyvale的Pharmacyclics制药公司与总部位于新泽西州Raritan的杨森生物科技公司共同销售。

识林-Kapok 2013-11-18

FDA approves Imbruvica for rare blood cancer
Second drug with breakthrough therapy designation to receive FDA approval
For Immediate Release: Nov. 13, 2013
Media Inquiries: Stephanie Yao, 301-796-0394, stephanie.yao@fda.hhs.gov
Consumer Inquiries: 888-INFO-FDA

The U.S. Food and Drug Administration today approved Imbruvica (ibrutinib) to treat patients with mantle cell lymphoma (MCL), a rare and aggressive type of blood cancer.

MCL is a rare form of non-Hodgkin lymphoma and represents about 6 percent of all non-Hodgkin lymphoma cases in the United States. By the time MCL is diagnosed, it usually has already spread to the lymph nodes, bone marrow and other organs.

Imbruvica is intended for patients with MCL who have received at least one prior therapy. It works by inhibiting the enzyme needed by the cancer to multiply and spread. Imbruvica is the third drug approved to treat MCL. Velcade (2006) and Revlimid (2013) are also approved to treat the disease.

“Imbruvica's approval demonstrates the FDA's commitment to making treatments available to patients with rare diseases,” said Richard Pazdur, M.D., director of the Office of Hematology and Oncology Products in the FDA's Center for Drug Evaluation and Research. “The agency worked cooperatively with the companies to expedite the drug's development, review and approval, reflecting the promise of the Breakthrough Therapy Designation program.”

Imbruvica is the second drug with breakthrough therapy designation to receive FDA approval. The Food and Drug Administration Safety and Innovation Act, passed in July 2012, gave the FDA the ability to designate a drug a breakthrough therapy at the request of the sponsor if preliminary clinical evidence indicates the drug may offer a substantial improvement over available therapies for patients with serious or life-threatening diseases.

The FDA is approving Imbruvica under the agency's accelerated approval program, which allows the FDA to approve a drug to treat a serious disease based on clinical data showing that the drug has an effect on a surrogate endpoint that is reasonably likely to predict a clinical benefit to patients. This program provides earlier patient access to promising new drugs while the company conducts confirmatory clinical trials. The FDA also granted Imbruvica priority review and orphan-product designation because the drug demonstrated the potential to be a significant improvement in safety or effectiveness in the treatment of a serious condition and is intended to treat a rare disease, respectively.

Imbruvica's accelerated approval for MCL is based on a study where 111 participants were given Imbruvica daily until their disease progressed or side effects became intolerable. Results showed nearly 66 percent of participants had their cancer shrink or disappear after treatment (overall response rate). An improvement in survival or disease-related symptoms has not been established.

The most common side effects reported in participants receiving Imbruvica are low levels of platelets in the blood (thrombocytopenia), diarrhea, a decrease in infection-fighting white blood cells (neutropenia), anemia, fatigue, musculoskeletal pain, swelling (edema), upper respiratory infection, nausea, bruising, shortness of breath (dyspnea), constipation, rash, abdominal pain, vomiting, and decreased appetite. Other clinically significant side effects include bleeding, infections, kidney problems and the development of other types of cancers.

Imbruvica is co-marketed by Sunnyvale, Calif.-based Pharmacyclics and Raritan, N.J.-based Janssen Biotech, Inc. Velcade (bortezomib) is marketed by Millennium Pharmaceuticals, based in Cambridge, Mass. Revlimid (lenalidomide) is marketed by Summit, N.J.-based Celgene.

原文请见：FDA NEWS RELEASE - FDA approves Imbruvica for rare blood cancer