欧洲药品管理局(EMA)已受理ixazomib用于复发/难治多发性骨髓瘤患者的市场营销授权申请(MAA)，ixazomib是口服蛋白酶体抑制剂类研究药物。
7月23日，ixazomib进入EMA人用药品委员会(CHMP)的加速审理，加速审理适用于具有重大公共卫生意义的药品，尤其是创新治疗药物。
武田法规事务副总裁Melody Brown说：“ixazomib在欧洲和美国的报批是我们预计在本财年末之前递交的若干申请中的首批申请。我们希望通过多地区快速连续报批，使尽可能多的复发/难治多发性骨髓瘤患者尽快受益于ixazomib。我们感谢全球参与TOURMALINE临床试验项目的患者和医生，他们的支持对呈报这些申请是至关重要的。”
MAA申请主要基于TOURMALINE-MM1试验的首次预设中期分析结果，该项枢纽性、国际性、随机、双盲、安慰剂对照3期临床试验纳入722例患者，旨在评估ixazomib联合来那度胺和地塞米松在复发/难治多发性骨髓瘤成人患者中是否优于安慰剂联合来那度胺和地塞米松。本试验中，患者接受持续治疗，直至疾病进展，并将评估远期转归。
ixazomib除了向EMA递交MAA申请外，还向美国食品药品管理局(FDA)递交了新药申请。向其他国家的申请计划于本财年下半年开始。
关于多发性骨髓瘤
多发性骨髓瘤是一种浆细胞癌，见于骨髓。多发性骨髓瘤中，一组浆细胞（或骨髓瘤细胞）转化为癌细胞并增生，使浆细胞的数目高于正常水平。由于浆细胞在体内广泛游走，有可能累及体内多数骨骼，可能导致压缩性骨折、骨溶解性病灶和相关疼痛。多发性骨髓瘤可导致若干严重健康问题，累及骨骼、免疫系统、肾脏和红细胞计数，部分较常见症状包括骨骼疼痛和疲乏，疲乏是贫血的症状。多发性骨髓瘤属罕见癌症，每年新发病例在欧盟约为39,000人、全球约为114,000人。
关于 ixazomib
ixazomib (MLN9708)是一种研究中的口服蛋白酶体抑制剂，正在研究用于多发性骨髓瘤、全身性轻链(AL)淀粉样变和其他恶性肿瘤。2011年，ixazomib在美国和欧洲被认定为用于多发性骨髓瘤的孤儿药，2012年，在美国和欧洲被认定为用于AL淀粉样变的孤儿药。2014年，ixazomib被美国食品药品管理局(FDA)认定为用于复发或难治AL淀粉样变的突破性治疗药物。它还是第一种进入3期临床试验的口服蛋白酶体抑制剂。
ixazomib 临床开发项目进一步巩固了武田为全球多发性骨髓瘤患者和治疗这些患者的医疗卫生专业人士开发创新治疗药物的不懈承诺。5项全球性3期试验正在进行中：
TOURMALINE-MM1研究ixazomib 联合来那度胺和地塞米松与安慰剂对照用于复发和/或难治多发性骨髓瘤
TOURMALINE-MM2研究ixazomib联合来那度胺和地塞米松与安慰剂对照用于新诊断的多发性骨髓瘤患者
TOURMALINE-MM3研究ixazomib与安慰剂对照用于新诊断的多发性骨髓瘤患者在诱导治疗和自体干细胞移植(ASCT)后的维持治疗
TOURMALINE-MM4研究ixazomib与安慰剂对照用于尚未接受ASCT的新诊断的多发性骨髓瘤患者的维持治疗
TOURMALINE-AL1研究ixazomib联合地塞米松与医师选用的治疗方案对照用于复发或难治AL淀粉样变
New Drugs Online Report for ixazomib
Information
Generic Name: ixazomib  
Trade Name:  
Synonym: MLN9708 
Entry Type: New molecular entity  
Development and Regulatory status
UK: Pre-registration (Filed) 
EU: Pre-registration (Filed) 
US: Pre-registration (Filed) 
UK launch Plans: Available only to registered users
Actual UK launch date:  
Comments
Aug 15: The EMA has accepted the Marketing Authorization Application for ixazomib for the treatment of patients with relapsed and/or refractory multiple myeloma. [12]
24/08/2015 09:52:51 
Jul 15: The EMA has granted an accelerated assessment to ixazomib for the treatment of patients with relapsed and/or refractory multiple myeloma. Filing is expected in the coming weeks. [11]
28/07/2015 10:20:35 
Jul 15: Filed in US for treatment of patients with relapsed and/or refractory multiple myeloma [10].
15/07/2015 16:01:14 
Jun 15: Has orphan status for MM in US [9].
29/06/2015 09:24:45 
Feb 15: Global filings are imminent [8].
11/02/2015 14:56:16 
Feb 14: Ixazomib remains PIII for MM [6].
10/03/2014 10:41:59 
Jun 12: Company report that the PIII study TOURMALINE-MM1 has started (NCT01564537) has started [2].
10/03/2014 10:39:10 
Sep 11: Granted orphan drug status in the EU (EU/3/11/899) [4].
07/10/2013 11:21:08 
Trial or other data
Feb 15: First interim analysis of the PIII, randomised, double-blind, placebo-controlled TOURMALINE-MM1 study of oral ixazomib in 722 pts with relapsed or refractory Multiple Myeloma met the primary endpoint of improvement in Progression-Free Survival (PFS). Pts treated with ixazomib + lenalidomide and dexamethasone lived without their disease worsening for a significantly longer time vs. pts who received placebo plus lenalidomide/dexamethasone [7]. 
11/02/2015 08:45:18
Jan 14: The global PIII TOURMALINE-MM1 study of ixazomib + lenalidomide and dexamethasone in patients with relapsed and/or refractory multiple myeloma has started in Asia (Australia, China, Japan, New Zealand, Singapore and South Korea). [5]
23/01/2014 09:27:54
Apr 12: NCT01564537 A PIII, randomized, double-blind, multicentre study comparing oral MLN9708 plus lenalidomide and dexamethasone vs placebo plus lenalidomide and dexamethasone in 703 adult patients with relapsed and/or refractory multiple myeloma who have received 1 to 3 prior therapies and an ECOG status 0-2. Primary outcome is progression free survival. Patients will receive 4mg MLN9708 (on days 1,8 and 1), 25mg lenalidomide (on days 1-21 and 40mg dexamethasone (on days 1,8, 15 and 22) every 28 days until disease progression. The study will start in Jun 12 and is due to complete Feb 19 (primary outcome data available from Jun 14) [1].
02/04/2012 14:57:12
Evidence Based Evaluations
NHSC/NIHR  http://www.hsc.nihr.ac.uk/topics/ixazomib-citrate-for-relapsed-and-or-refractory-mu/ 
References  
Available only to registered users
 Category
BNF Category: Other antineoplastic drugs (08.01.05)
Pharmacology: Proteasome inhibitor  
Epidemiology: The UK incidence of MM is 7.7 per 100,000 people and prevalence is about 16 in 100,000 people.  
Indication: Multiple myeloma (MM) 
Additional Details: relapsed and/or refractory, combination therapy 
Method(s) of Administration  
Oral 
Company Information
Name: Takeda 
US Name: Millennium Pharmaceuticals 
Further Information
Anticipated commissioning route (England) NHSE 
High cost drug list? Awaiting Update
Implications Available only to registered users