阿杜卡奴单抗治疗阿尔茨海默病：监管视角。

Aducanumab for Alzheimer's disease: A regulatory perspective.

机构信息

School of Pharmacy, Department of Biology, University of Tor Vergata, Rome, Italy; Laboratory of Pharmacology of Synaptic Plasticity, Fondazione EBRI Rita Levi Montalcini, Rome, Italy.

School of Pharmacy, Department of Biology, University of Tor Vergata, Rome, Italy; Malta Medicines Authority, Malta Life Sciences Park, San Ġwann SĠN 3000, Malta.

出版信息

Pharmacol Res. 2021 Sep;171:105754. doi: 10.1016/j.phrs.2021.105754. Epub 2021 Jul 2.

DOI:10.1016/j.phrs.2021.105754

PMID:34217830

Abstract

On June 7th 2021, the Food and Drug Administration (FDA) granted approval for Aduhelm (aducanumab) for the treatment of Alzheimer's disease under its accelerated approval program. Aducanumab is the first putative disease-modifying therapy (DMT) approved for the treatment of AD with a great potential for clinical benefit over current symptomatic therapies. The scientific community has been largely confounded by this historical decision since this has been based on the reduction of a surrogate marker (amyloid beta) and not on data showing clinical efficacy. Here we provide a regulatory perspective on the topic and discuss potential similarities and differences between the FDA's and EMA's evaluative processes.

摘要

2021 年 6 月 7 日，美国食品和药物管理局（FDA）通过加速审批程序批准 aducanumab（阿杜那单抗）用于治疗阿尔茨海默病。Aducanumab 是首个获批的潜在疾病修正疗法（DMT），与目前的对症治疗相比，具有很大的临床获益潜力。由于这一历史性决策是基于替代标志物（β淀粉样蛋白）的减少，而不是基于显示临床疗效的数据，科学界对此感到非常困惑。在这里，我们从监管角度探讨了这一话题，并讨论了 FDA 和 EMA 评估过程之间的潜在相似性和差异。