美東時間6月7日周一，美國食品和藥物管理局（FDA）批準了渤?。˙iogen）治療阿爾茨海默病的試驗性藥物aducanumab，該藥品將以Aduhelm作為名稱上市。
英國《金融時報》報道，這種藥物可以清除患者大腦中形成的粘性結塊，一些科學家認為，正是這種斑塊導致了阿爾茨海默癥。迄今為止，其它已獲批準的阿爾茨海默癥藥物都是為了治療癥狀，而非減緩或逆轉(zhuǎn)疾病進程。Aduhelm是首個被批準旨在減緩阿爾茨海默癥病情發(fā)展的藥物。
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.
藥品獲得“空前關注”
鑒于全球?qū)Π柎暮Ｄ∷幬锏钠惹行枨?，Biogen上周五收盤股價大漲5%，周一盤前股價繼續(xù)上漲近5%，目前股價觸及300美元。
FDA藥物評估和研究中心主任Patrizia Cavazzoni博士在一份新聞稿中指出：
我們很清楚有許多人都在關注這一（藥品）的批準。我們知道媒體、阿爾茨海默病患者群體、官員以及其他利益相關方都在關心這件事。針對阿爾茨海默這一危及生命的病癥尚未有明確的治療方案，因此這次的審查結果得到如此多人的關注是很正常的。
'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.
藥品售價及其有效性
成市場關注焦點
售價方面，aducanumab是一種靜脈注射藥物，每年的療程費用預計為5.6萬美元，每針為4,312美元。CNBC表示，市場預計，最新藥物的批準將為公司帶來數(shù)十億美元的收入。
不過需要注意的是，F(xiàn)DA本次批準Aduhelm并非完全無條件，而是需要進行“第四階段”臨床研究。對此FDA表示，渤健提交的材料中來自于兩項三期臨床試驗的數(shù)據(jù)非常復雜，而且仍有不確定性殘留。
部分臨床醫(yī)生對此藥物同樣抱有懷疑態(tài)度。據(jù)CNBC，部分醫(yī)生表示，由于支撐Aduhelm的臨床數(shù)據(jù)喜憂參半，如果該藥品真的上市，他們不會在處方中開出此藥。
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.
什么是阿爾茨海默癥？
阿爾茨海默癥是一種不可逆的漸進性腦部疾病，發(fā)病進程緩慢，主要影響記憶和其他心智能力，最終導致患者生活無法自理。
阿爾茨海默癥是老年人最常見的一種癡呆癥，但不完全等同于老年癡呆癥（Dementia），癡呆癥可由許多不同的醫(yī)學病癥（如嚴重的頭部損傷或大中風）引起。
目前全世界至少有5000萬的癡呆患者，到2050年預計將達到1.52億，其中約60%-70%為阿爾茨海默病患者。
阿爾茲海默病的治療難點在于其發(fā)病機制并未完全清晰。目前，比較公認的機制是認為β淀粉樣蛋白（ amyloid-β，Aβ） 的生成和清除失衡是神經(jīng)元變性和癡呆發(fā)生的始動因素，異常水平的β-淀粉樣蛋白在大腦神經(jīng)元之間形成的斑塊具有神經(jīng)毒性，導致神經(jīng)元變性。
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.
