2015年5月8日,艾伯维公司开发的治疗白血病新药venetoclax获得了FDA的突破性药物认证。FDA认为这种药物在治疗带有17p基因缺失突变的慢性粒细胞白血病患者方面有着显著疗效。数据显示，目前约有3%-10%的一线慢性粒细胞白血病患者都带有这种基因突变，而在出现抗药性的慢性白血病患者中，这一比例更是高达50%之多。
在去年年底召开的美国血液学会年会（ASH）上，venetoclax就曾以其出色的疗效令分析人士印象深刻。在一项由慢性粒细胞白血病复发患者和抗药性患者参与的临床研究中，患者在使用了venetoclax和Rituxan联合治疗方案后，总体缓解率达到了88%之多，正是基于这些出色数据，FDA才认为venetoclax匹配得上突破性药物这一殊荣。认为这一药物有成为白血病顶级疗法的潜质。
Venetoclax目前由艾伯维公司和罗氏公司联合开发。该药物是一种实验性B细胞淋巴瘤因子-2(BCL-2)抑制剂。BCL-2是一种可阻止一些细胞（包括淋巴细胞）凋亡的蛋白，该因子在发生于淋巴结、批准和免疫系统其他器官中的癌细胞高度表达。venetoclax旨在选择性抑制BCL-2因子的功能，恢复细胞的通讯系统，让癌细胞自我毁灭，以达到治疗肿瘤的效果
New Drugs Online Report for venetoclax
Information
Generic Name: venetoclax  
Trade Name:  
Synonym: GDC-0199, ABT-199, RG7601 
Entry Type: New molecular entity  
Development and Regulatory status
UK: Phase III Clinical Trials 
EU: Phase III Clinical Trials 
US: Phase III Clinical Trials 
UK launch Plans: Available only to registered users
Actual UK launch date:  
Comments
May 15: FDA grant Breakthrough Therapy Designation [5].
08/05/2015 09:13:08 
Mar 14: EU & US filings planned for 2016 [3].
23/06/2014 10:45:40 
Trial or other data
Q1 14: First pt enrolled in PIII MURANO as planned [4].
16/03/2015 10:42:28
Jun 14: PIII NCT02005471 = MURANO. First pt expected to be enrolled Q1 14 [3].
23/06/2014 11:09:12
Jun 14: At the interim point of a PIb study, ABT-199/GDC-0199 combined with rituximab registered an 84% overall response rate among patients with CLL. Nine of 25 evaluable patients in the study achieved either a complete response or complete response with incomplete blood count recovery, while close to half--12 patients--achieved a partial response. Among the complete responders, 75% (6 patients) were identified as minimal residual disease negative, indicating the detection of zero leukaemic cells [2].
09/06/2014 12:50:00
Roche arecollaborating with AbbVie on development.
10/12/2013 10:22:42
NCT02005471 is a multicentre, PIII, open-label, randomized study in r370 elapsed/refractory patients with CLL of GDC-0199 (ABT-199) + rituximab vs bendamustine + rituximab. Patients randomized to GDC-0199+R will be given GDC-0199 daily (oral, target dose 400 mg) until disease progression or 2 years since treatment start, whichever comes first. and will receive 6 cycles of IV rituximab. Patients randomized to B+R will receive 6 cycles of treatment. Anticipated time on study is up to 5 years. The primary outcome is PFS. The study starts Dec 13 and is due to complete Aug 20 [1].
10/12/2013 10:15:28 
References  
Available only to registered users
 Category
BNF Category: Other antineoplastic drugs (08.01.05)
Pharmacology: Novel small molecule that selectively blocks the function of Bcl-2 proteins and with the goal of enhancing apoptosis. Bcl-2 proteins are expressed at high levels in non-Hodgkin lymphoma (NHL), CLL and in other B-cell neoplasms.  
Epidemiology: CLL is the most common leukaemia in the Western world with an incidence of 4.2/100,000/year. The incidence increases to >30/100,000/year at an age of >80 years. Median age at diagnosis is 72 years. About 10% of CLL patients are reported to be younger than 55 years.  
Indication: Chronic lymphocytic leukaemia (CLL) 
Additional Details: relapsed/refractory, combination therapy 
Method(s) of Administration  
Oral 
Company Information
Name: Roche 
US Name: Roche 
Further Information
Anticipated commissioning route (England) NHSE 
High cost drug list? Awaiting Update
Implications Available only to registered users