——新型抗癌药Venclexta（venetoclax）突破性治疗慢性淋巴细胞性白血病
2016年4月11日,美国食品和药品监管局(FDA)批准Venclexta(venetoclax)为有慢性淋巴细胞性白血病(CLL)有一个染色体异常称为17p缺失和曽被至少一种以前治疗患者的治疗。Venclexta是第一个靶向B-细胞淋巴瘤2(BCL-2)蛋白被FDA-批准治疗，BCL-2支持癌细胞生长和在许多有CLL患者过表达。.
按照美国癌症研究所，CLL是在成年白血病最常见类型之一，每年约有15,000新病例被诊断。CLL特征是异常淋巴细胞进行性积蓄，一种类型白细胞。有CLL患者有一种17p缺失缺失作用于抑制癌生长的染色体一部分。这个染色体异常发生在约10%未治疗过CLL患者和在约20%复发的CLL患者。
FDA的药品评价和研究中心中血液学和肿瘤室主任Richard Pazdur 说：“这些患者现有一种新，抑制涉及保持肿瘤细胞存活蛋白的靶向治疗，” “对某些用其他治疗没有有利结局的CLL有患者，Venclexta对他们的特殊情况可能提供一个新选择。”
在一项单-臂临床试验106例有CLL有一个17p缺失和患者曽接受至少一种以前治疗患者测试Venclexta的疗效。试验参加者每天口服Venclexta，开始用20 mg和在历时五周阶段增加至400 mg，结果显示80%试验参加者经历他们的癌症一个完全或部分缓解。
Venclexta是适用为在使用FDA-批准的协同诊断Vysis CLL FISH探针药盒确证17p缺失的检测后患者的每天使用。
Venclexta的最常见副作用包括中性粒细胞减少，腹泻，恶心，贫血，上呼吸道感染，血小板减少和疲乏。严重并发症可能包括肺炎，发热性中性粒细胞减少，发热，自身免疫溶血性贫血，贫血和代谢异常被称为肿瘤溶解综合证。用Venclexta患者不应给予减毒活疫苗.
FDA授权Venclexta申请对此适应证突破性治疗指定，优先审评 状态，和加速批准。这些是不同的程序意向促进和加快鉴于它们对有严重或危及生命情况患者潜在获益的某些药物开发和审评。 Venclexta还接受孤儿药物指定，它提供奖励例如税收减免，用户费用减免和eligibility 对专有权资格有助于和鼓励对罕见疾病药物的开发。
Venclexta是由伊利诺伊北芝加哥的AbbVie公司制造，和加州旧金山的AbbVie和Genentech 美国公司上市。Vysis CLL FISH探针药盒由伊利诺伊Des Plaines的Abbott Molecular制造.

Venclexta, venetoclax (ABT-199, RG7601) (formerly GDC-0199)
Important Safety Information
Strong CYP3A inhibitors, a certain type of medicine, must not be taken when patients first start taking Venclexta and while their dose is being slowly increased.
Patients must tell their doctor right away about any side effects they experience.
Venclexta can cause serious side effects, including tumor lysis syndrome (TLS). TLS is caused by the fast breakdown of cancer cells. TLS can cause kidney failure, the need for dialysis treatment, and may lead to death. A patient's doctor will do tests for TLS. It is important for patients taking Venclexta to keep their appointments for blood tests. Patients will receive other medicines before starting and during treatment with Venclexta to help reduce their risk of TLS. Patients may also need to receive intravenous (IV) fluids into their vein. Patients taking Venclexta should tell their doctor right away if they have any symptoms of TLS during treatment with Venclexta, including fever, chills, nausea, vomiting, confusion, shortness of breath, seizures, irregular heartbeat, dark or cloudy urine, unusual tiredness or muscle pain or joint pain.
Patients should drink plenty of water when taking Venclexta to help reduce the risk of getting TLS. Patients should drink 6 to 8 glasses (about 56 ounces total) of water each day, starting 2 days before their first dose, on the day of their first dose of Venclexta, and each time the dose is increased.
Certain medicines must not be taken when patients first start taking Venclexta and while their dose is being slowly increased.
■Patients should tell their doctor about all the medicines they take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Venclexta and other medicines may affect each other, causing serious side effects.
■Patients should not start new medicines during treatment with Venclexta without first talking with their doctor.
Before taking Venclexta, patients should tell their doctor about all of their medical conditions, including if they:
■Have any kidney or liver problems.
■Have problems with their body salts or electrolytes, such as potassium, phosphorus, or calcium.
■Have a history of high uric acid levels in their blood or gout.
■Are scheduled to receive a vaccine. Patients should not receive a "live vaccine" during or after treatment with Venclexta.
■Are pregnant or plan to become pregnant. Venclexta may harm an unborn baby. If a patient is able to become pregnant, the doctor should do a pregnancy test before they start treatment with Venclexta, and they should use effective birth control during treatment and for 30 days after the last dose of Venclexta.
■Are breastfeeding or plan to breastfeed. It is not known if Venclexta passes into breast milk. Patients should not breastfeed during treatment with Venclexta.
Patients taking Venclexta should not drink grapefruit juice or eat grapefruit, Seville oranges (often used in marmalades), or starfruit while they are taking Venclexta. These products may increase the amount of Venclexta in the patient's blood.
Venclexta can cause serious side effects, including:
■Low White Blood Cell Count (neutropenia): Low white blood cell counts are common with Venclexta, but can also be severe. A doctor will do blood tests to check a patient's blood counts during treatment with Venclexta. Patients should tell their doctor right away if they have a fever or any signs of an infection.
The most common side effects of Venclexta include diarrhea, nausea, low red blood cell count, upper respiratory tract infection, low platelet count, and feeling tired.
Venclexta may cause fertility problems in males. This may affect the ability to father a child. Patients should talk to their doctor if they have concerns about fertility.
These are not all the possible side effects of Venclexta. Patients should tell their doctor if they have any side effect that bothers them or that does not go away.
http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm495253.htm