OnabotulinumtoxinA (BTX-A) was first linked to beneficial effects in migraine 25 years ago and was approved by the FDA for preventive treatment of chronic migraine in 2010. The treatment has since had a major impact on the well-being of people with chronic migraine. The clinical development programme for BTX-A and research since its approval have provided insights into the neuromodulatory sensory effect of BTX-A, how it can control chronic migraine despite its peripheral action, and the underlying biology of migraine as a disease. In this Review, we consider the impact that BTX-A has had on the management of chronic migraine and on the research field. We discuss the insights provided by clinical research, encompassing the clinical trials and subsequent real-world evidence, and the mechanistic insights provided by preclinical and translational research. We also provide an overview of future directions of research in the field BTX-A in migraine and the clinical translation of this research.

OnabotulinumtoxinA (BTX-A) 于 25 年前首次与偏头痛相关，并于 2010 年被 FDA 批准用于慢性偏头痛的预防性治疗。此后，该治疗对慢性偏头痛患者的健康产生了重大影响。 BTX-A 的临床开发计划和自批准以来的研究深入了解了 BTX-A 的神经调节感觉作用、它如何控制慢性偏头痛（尽管其外周作用）以及偏头痛作为一种疾病的潜在生物学。在这篇综述中，我们考虑了 BTX-A 对慢性偏头痛治疗和研究领域的影响。我们讨论临床研究提供的见解，包括临床试验和随后的现实世界证据，以及临床前和转化研究提供的机制见解。我们还概述了 BTX-A 治疗偏头痛领域的未来研究方向以及该研究的临床转化。