Ricklin Daniel
Molecular Pharmacy Group, Department of Pharmaceutical Sciences, University of Basel, Basel, Switzerland.
Eur J Immunol. 2024 Dec;54(12):e2350816. doi: 10.1002/eji.202350816. Epub 2024 Sep 12.
Therapeutic interventions in the complement system, a key immune-inflammatory mediator and contributor to a broad range of clinical conditions, have long been considered important yet challenging or even unfeasible to achieve. Almost 20 years ago, a spark was lit demonstrating the clinical and commercial viability of complement-targeted therapies. Since then, the field has experienced an impressive expansion of targeted indications and available treatment modalities. Currently, a dozen distinct complement-specific therapeutics covering several intervention points are available in the clinic, benefiting patients suffering from eight disorders, not counting numerous clinical trials and off-label uses. Observing this rapid rise of complement-targeted therapy from obscurity to mainstream with amazement, one might ask whether the peak of this development has now been reached or whether the field will continue marching on to new heights. This review looks at the milestones of complement drug discovery and development achieved so far, surveys the currently approved drug entities and indications, and ventures a glimpse into the future advancements yet to come.
补体系统是一种关键的免疫炎症介质，与多种临床病症相关。长期以来，针对补体系统的治疗干预一直被认为很重要，但实现起来具有挑战性甚至不可行。大约20年前，出现了一丝曙光，证明了补体靶向疗法在临床和商业上的可行性。从那时起，该领域在靶向适应症和可用治疗方式方面取得了令人瞩目的扩展。目前，临床上有十几种针对补体的独特疗法，涵盖多个干预点，使八种疾病的患者受益，这还不包括众多的临床试验和超说明书用药情况。惊叹于补体靶向疗法从默默无闻迅速崛起为主流疗法，人们可能会问，这一发展是否已达到顶峰，或者该领域是否会继续迈向新的高度。本文回顾了迄今为止补体药物发现和开发的里程碑，调查了目前已获批的药物实体和适应症，并展望了未来的发展。
