美国FDA批准治疗阿尔茨海默症药物 药品售价及有效性成争议话题
美东时间6月7日周一，美国食品和药物管理局（FDA）批准了渤健（Biogen）治疗阿尔茨海默病的试验性药物aducanumab，该药品将以Aduhelm作为名称上市。
英国《金融时报》报道，这种药物可以清除患者大脑中形成的粘性结块，一些科学家认为，正是这种斑块导致了阿尔茨海默症。迄今为止，其它已获批准的阿尔茨海默症药物都是为了治疗症状，而非减缓或逆转疾病进程。Aduhelm是首个被批准旨在减缓阿尔茨海默症病情发展的药物。
The U.S. Food and Drug Administration (FDA) approved Aduhelm (aducanumab) on Monday for the treatment of Alzheimer's, the first new medicine against the disease in almost two decades and the first to address cognitive decline linked to the condition.
Aduhelm was approved using the accelerated approval pathway, which can be used for a drug for a serious or life-threatening illness that provides a meaningful therapeutic advantage over existing treatments, according to the FDA.
药品获得“空前关注”
鉴于全球对阿尔茨海默病药物的迫切需求，Biogen上周五收盘股价大涨5%，周一盘前股价继续上涨近5%，目前股价触及300美元。
FDA药物评估和研究中心主任Patrizia Cavazzoni博士在一份新闻稿中指出：
我们很清楚有许多人都在关注这一（药品）的批准。我们知道媒体、阿尔茨海默病患者群体、官员以及其他利益相关方都在关心这件事。针对阿尔茨海默这一危及生命的病症尚未有明确的治疗方案，因此这次的审查结果得到如此多人的关注是很正常的。
'Aduhelm is the first treatment directed at the underlying pathophysiology of Alzheimer's disease, the presence of amyloid-beta plaques in the brain,' said Dr. Patrizia Cavazzoni, director of the FDA's Center for Drug Evaluation and Research.
'We are well-aware of the attention surrounding this approval,' Cavazzoni said in a statement. 'We understand that Aduhelm has garnered the attention of the press, the Alzheimer's patient community, our elected officials, and other interested stakeholders.'
'With a treatment for a serious, life-threatening disease in the balance, it makes sense that so many people were following the outcome of this review,' Cavazzoni added.
药品售价及其有效性
成市场关注焦点
售价方面，aducanumab是一种静脉注射药物，每年的疗程费用预计为5.6万美元，每针为4,312美元。CNBC表示，市场预计，最新药物的批准将为公司带来数十亿美元的收入。
不过需要注意的是，FDA本次批准Aduhelm并非完全无条件，而是需要进行“第四阶段”临床研究。对此FDA表示，渤健提交的材料中来自于两项三期临床试验的数据非常复杂，而且仍有不确定性残留。
部分临床医生对此药物同样抱有怀疑态度。据CNBC，部分医生表示，由于支撑Aduhelm的临床数据喜忧参半，如果该药品真的上市，他们不会在处方中开出此药。
The FDA said it will continue to monitor the drug as it reaches the U.S. market. The FDA granted the approval on the condition that Biogen, the manufacturer, conduct a new trial. The Massachusetts-based biotechnology company said on Monday that aducanumab's list price is $56,000 per year and $4,312 per infusion.
The FDA's approval of Aduhelm is not unconditional, but requires a Phase IV clinical study. Last November, a panel of outside experts that advises the FDA unexpectedly declined to endorse the experimental drug, citing unconvincing data. It also criticized FDA staff for what it called an overly positive review.
什么是阿尔茨海默症？
阿尔茨海默症是一种不可逆的渐进性脑部疾病，发病进程缓慢，主要影响记忆和其他心智能力，最终导致患者生活无法自理。
阿尔茨海默症是老年人最常见的一种痴呆症，但不完全等同于老年痴呆症（Dementia），痴呆症可由许多不同的医学病症（如严重的头部损伤或大中风）引起。
目前全世界至少有5000万的痴呆患者，到2050年预计将达到1.52亿，其中约60%-70%为阿尔茨海默病患者。
阿尔兹海默病的治疗难点在于其发病机制并未完全清晰。目前，比较公认的机制是认为β淀粉样蛋白（ amyloid-β，Aβ） 的生成和清除失衡是神经元变性和痴呆发生的始动因素，异常水平的β-淀粉样蛋白在大脑神经元之间形成的斑块具有神经毒性，导致神经元变性。
Alzheimer's disease is a progressive neurodegenerative disorder that slowly destroys memory and cognitive skills. According to the Alzheimer's Association, more than 6 million Americans are living with the disease. The group estimates that number will climb to 13 million by 2050.