【国际新药】FDA授予masitinib（马赛替尼）治疗肌萎缩侧索硬化症孤儿药地位
2015/3/24 经纬传奇
专业从事蛋白激酶抑制剂研发及销售的AB Science公司近日宣布，FDA已授予masitinib(马赛替尼)治疗肌萎缩侧索硬化症(ALS)的孤儿药地位。目前，AB Science正在一项III期临床中调查masitinib治疗ALS的潜力。今年1月，一个外部数据和安全监测委员会(DSMB)根据最新安全性数据的审查结果，建议该项研究继续推进。
去年火爆全球的"冰桶挑战"便是一个呼吁全社会关注ALS(肌萎缩侧索硬化症)等罕见病的公益性活动。ALS(Amyotrophic lateral sclerosis)是一种渐进性神经退行性疾病，该病影响大脑和脊髓中与运动相关的神经细胞，造成运动神经元死亡，令大脑无法控制肌肉运动，而肌肉也会因缺乏运动而萎缩。ALS是一种罕见病，发病率约十万分之二到十万分之五。美国约有3万人确诊，欧洲约1万5千人。
ALS初期症状包括肌肉无力或行动僵硬，可能伴随肌肉萎缩，多数从手臂或腿部无力开始，也可能伴有吞咽困难。在疾病晚期，ALS患者会完全失去行动能力。ALS确诊后患者预期寿命约3-5年，但也有约20%病人可以存活超过5年，5%的病人存活超过20年(著名物理学家霍金就是5%的一员，他已经与该病斗争了40多年)。
masitinib是一种新型口服给药的酪氨酸激酶抑制剂，通过抑制有限数量的激酶靶向肥大细胞和巨噬细胞。基于其独特的作用机制，masitinib具有开发用于肿瘤、炎症性疾病、中枢神经系统等多种疾病的潜力。
英文原文：AB Science: Masitinib Receives Orphan Drug Designation for Amyotrophic Lateral Sclerosis from FDA
PARIS, March 20, 2015 (GLOBE NEWSWIRE) -- AB Science SA (NYSE Euronext - FR0010557264 - AB), a pharmaceutical company specialized in research, development and marketing of protein kinase inhibitors (PKIs), announces that the U.S. Food and Drug Administration (FDA) has granted the company Orphan Drug designation for masitinib in the treatment of amyotrophic lateral sclerosis.
The FDA's Office of Orphan Drug Products Development reviews applications for Orphan Drug status to support development of medicines for underserved patient populations, or rare disorders that affect fewer than 200,000 people in the United States. The successful application submitted by AB Science and the FDA granting of Orphan Drug status entitles the company to a seven-year period of marketing exclusivity in the United States for masitinib, if it is approved by the FDA for the treatment of amyotrophic lateral sclerosis. Orphan Drug status also enables the company to apply for research grant funding for Phase I and II Clinical Trials, tax credits for certain research expenses, and a waiver from the FDA's application user fee, as well as additional support from FDA and a potentially faster regulatory process.
A phase 3 is currently on-going with masitinib in ALS. In January 2015, the external Data and Safety Monitoring Board (DSMB) recommended the continuation of this phase 3 study based upon review of the latest safety data. The DSMB was created as part of the Company's pivotal clinical study evaluating masitinib in the treatment of amyotrophic lateral sclerosis.
Amyotrophic lateral sclerosis is a rare degenerative disorder that results in progressive wasting and paralysis of voluntary muscles. There are approximately 30,000 people with ALS in the European unio and 15,000 in the US, with more than 7,500 new cases diagnosed each year in Europe and 4,500 in the US. Almost 50% of ALS patients die within 3 years and 90% die within 5 years.
about Orphan Drug Designation
The FDA Office of Orphan Products Development (OOPD) mission is to advance the evaluation and development of products (drugs, biologics, devices, or medical foods) that demonstrate promise for the diagnosis and/or treatment of rare diseases or conditions. In fulfilling that task, OOPD evaluates scientific and clinical data submissions from sponsors to identify and designate products as promising for rare diseases and to further advance scientific development of such promising medical products.
The approval of an orphan designation request does not alter the standard regulatory requirements and process for obtaining marketing approval for investigational use. Sponsors must establish safety and efficacy of a compound in the treatment of a disease through adequate and well-controlled studies. However, the FDA review process may be speedier for Orphan Drugs than those which do not receive Orphan Drug designation.
about masitinib
Masitinib is a new orally administered tyrosine kinase inhibitor that targets mast cells and macrophages, important cells for immunity, through inhibiting a limited number of kinases. based on its unique mechanism of action, masitinib can be developed in a large number of conditions in oncology, in inflammatory diseases, and in certain diseases of the central nervous system. In oncology due to its immunotherapy effect, masitinib can have an effect on survival, alone or in combination with chemotherapy. Through its activity on mast cells and consequently the inhibition of the activation of the inflammatory process, masitinib can have an effect on the symptoms associated with some inflammatory and central nervous system diseases and the degeneration of these diseases.
about AB Science
Founded in 2001, AB Science is a pharmaceutical company specializing in the research, development and commercialization of protein kinase inhibitors (PKIs), a class of targeted proteins whose action are key in signaling pathways within cells. Our programs target only diseases with high unmet medical needs, often lethal with short term survival or rare or refractory to previous line of treatment in cancers, inflammatory diseases, and central nervous system diseases, both in humans and animal health.
AB Science has developed a proprietary portfolio of molecules and the Company's lead compound, masitinib, has already been registered for veterinary medicine in Europe and in the USA. The company is currently pursuing thirteen phase 3 studies in human medicine in first-line and second-line GIST, metastatic melanoma expressing JM mutation of c-Kit, multiple myeloma, metastatic colorectal cancer, metastatic prostate cancer, pancreatic cancer, mastocytosis, severe persistent asthma, rheumatoid arthritis, Alzheimer's disease, progressive forms of multiple sclerosis, and Amyotrophic Lateral Sclerosis. The company is headquartered in Paris, France, and listed on Euronext Paris 。(来源：生物谷)
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