开年便遭遇当头一棒，曾被业界视为超重量级重磅口服降糖药fasiglifam因安全性问题惨遭失败；今年4月，与礼来联合推广糖尿病药物艾可拓（Actos）因隐瞒膀胱癌风险遭遇90亿美元天价罚单，不过该罚单在今年10月发生惊天大逆转降至3680万美元，让武田和礼来着实地经历了一场过山车般的惊险刺激。
然而，武田在2014年也取得了一些胜利，其中最瞩目的是单抗药物Entyvio（vedolizumab）今年5月拿下美欧2大市场，同时肿瘤学药物万珂（Velcade）扩大适应症也获得了FDA批准。近日，武田寄予厚望的罕见病药物ixazomib也传来了好消息，FDA已授予ixazomib（MLN9708）突破性疗法认定。业界预测，ixazomib的前景将超越武田推出的市面最成功的多发性骨髓瘤药物Velcade（万珂），该药是过去11年中唯一被证明能够延缓多发性骨髓瘤患者总生存期（OS）的药物。
ixazomib是一种口服蛋白酶体抑制剂，开发用于复发性或难治性系统性轻链型淀粉样变性（ALamyloidosis）的治疗。该药是FDA授予突破性疗法认定的首个蛋白酶体抑制剂和AL型淀粉样变性实验性药物。
根据武田，AL型淀粉样变性是一种罕见、侵略性蛋白质错误折叠疾病。在美国，每年确诊AL型淀粉样变性的患者总数少于3000例。目前，武田淀粉样变性（amyloidosis）项目的推进堪称神速，直接从I期跳至III期。不过，该领域武田也有对手，爱尔兰生物技术公司Prothena也正在开发一种治疗淀粉样变性的药物，目前处于较早期阶段。
英文原文：Takeda’s Investigational, Oral Proteasome Inhibitor Ixazomib Granted Breakthrough Therapy Designation by U.S. FDA for Relapsed or Refractory Systemic Light-chain Amyloidosis
First proteasome inhibitor and first investigational therapy for amyloidosis to receive FDA Breakthrough Therapy designation
CAMBRIDGE, Mass. & OSAKA, Japan--(BUSINESS WIRE)--
Takeda Pharmaceutical Company Limited (TSE:4502) today announced that the U.S. Food & Drug Administration (FDA) has granted Breakthrough Therapy status to the company’s investigational, oral proteasome inhibitor, ixazomib (MLN9708), for the treatment of relapsed or refractory systemic light-chain (AL) amyloidosis. This is the first proteasome inhibitor and first investigational therapy for AL amyloidosis to receive Breakthrough Therapy designation.
The development program for ixazomib in this indication progressed directly from a Phase 1 to a Phase 3 clinical trial, TOURMALINE-AL1, which is currently evaluating ixazomib plus dexamethasone in patients with relapsed or refractory AL amyloidosis. This is the only Phase 3 trial for relapsed or refractory AL amyloidosis and it is recruiting globally.
Breakthrough Therapy designation is intended to expedite the development and review of new medicines to treat serious or life-threatening conditions. Breakthrough Therapy designation requires preliminary clinical evidence indicating that the drug may demonstrate substantial improvement on a clinically significant endpoint(s) over available therapies. The data used to support this designation will be presented at the American Society of Hematology (ASH) annual meeting to be held December 6-9, 2014 in San Francisco [Abstract 3450: Long-Term Outcome of a Phase 1 Study of the Investigational Oral Proteasome Inhibitor (PI) Ixazomib at the Recommended Phase 3 Dose (RP3D) in Patients (Pts) with Relapsed or Refractory Systemic Light-Chain (AL) Amyloidosis (RRAL)].
“Patients with AL amyloidosis face a debilitating disease that can affect many of their organs and tissues. The Breakthrough Therapy designation for ixazomib is a major milestone in the development of new treatment options for patients battling this rare and aggressive disease,” said Raymond L. Comenzo, M.D., Director, Blood Bank and Stem Cell Processing Laboratory and Professor, Tufts University School of Medicine. “We are encouraged by positive preliminary data evaluating use of ixazomib in the treatment of patients with relapsed or refractory AL amyloidosis.”
“Ixazomib is a breakthrough drug showing activity and organ improvement in most of these heavily pretreated AL amyloidosis patients,” said Professor Giampaolo Merlini, Director, Center for Research and Treatment of Systemic Amyloidosis, University of Pavia, Italy.
AL amyloidosis is a rare and aggressive protein misfolding disorder with fewer than 3,000 cases diagnosed in the U.S. every year. It is characterized by the deposition of amyloid in bodily organs and tissues. While AL amyloidosis can affect different organs in different people, it frequently affects the heart, kidneys, liver, spleen, nervous system, and gastrointestinal tract. There are no approved treatments in the U.S. or globally for AL amyloidosis, representing a significant unmet medical need.
“This Breakthrough Therapy designation is also an important recognition of the strength of our oncology development program and our commitment to extend proteasome inhibition to conditions that have not traditionally been the focus of research and development,” said Michael Vasconcelles, M.D., Head, oncology Therapeutic Area Unit, Takeda. “We would like to acknowledge and thank the patients, study investigators, and institutions whose participation in this program have made this milestone possible.”
Compounds that receive Breakthrough Therapy status receive more intensive FDA guidance on an efficient drug development program and an enhanced agency commitment of senior personnel.
about Ixazomib
Ixazomib is an investigational oral proteasome inhibitor, which is being studied in multiple myeloma (MM), systemic light-chain (AL) amyloidosis and other malignancies. Ixazomib was granted orphan drug designation in MM in both the U.S. and Europe in 2011, and for AL amyloidosis in both the U.S. and Europe in 2012. It is the first oral proteasome inhibitor to enter Phase 3 clinical trials. Four global Phase 3 trials are ongoing: TOURMALINE-MM1, investigating ixazomib vs. placebo in combination with lenalidomide and dexamethasone in relapsed and/or refractory MM; TOURMALINE-AL1, investigating ixazomib plus dexamethasone in patients with relapsed or refractory AL amyloidosis; TOURMALINE-MM2, investigating ixazomib vs. placebo in combination with lenalidomide and dexamethasone in patients with newly diagnosed MM; and TOURMALINE-MM3, investigating ixazomib vs. placebo as maintenance therapy in patients with newly diagnosed MM following induction therapy and autologous stem cell transplant. For additional information on the ongoing Phase 3 studies please visit www.tourmalinetrials.com or www.clinicaltrials.gov.
about Amyloidosis
Amyloidosis is a disease that occurs when the body's antibody-producing cells do not function properly and produce abnormal protein fibers known as amyloids. These amyloids can form deposits in any organ in the body. For this reason, amyloidosis can affect people differently. There are different types of amyloidosis and some are inherited. AL amyloidosis, also known as light-chain or primary or systemic amyloidosis, is the most common form of the disease. AL amyloidosis most frequently affects the kidneys and heart. However, almost any other tissue can be affected, including the liver, nervous system, and soft tissues.
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