Activating mutations in the FGFR3 receptor tyrosine kinase lead to most prevalent form of genetic dwarfism in humans, the achondroplasia. Many features of the complex function of FGFR3 in growing skeleton were characterized, which facilitated identification of therapy targets, and drove progress toward treatment. In August 2021, the vosoritide was approved for treatment of achondroplasia, which is based on a stable variant of the C-natriuretic peptide. Other drugs may soon follow, as several conceptually different inhibitors of FGFR3 signaling progress through clinical trials. Here, we review the current achondroplasia therapeutics, describe their mechanisms, and illuminate motivations leading to their development. We also discuss perspectives of curing achondroplasia, and options for repurposing achondroplasia drugs for dwarfing conditions unrelated to FGFR3.

FGFR3受体酪氨酸激酶的激活突变导致人类最普遍的遗传性侏儒症，即软骨发育不全。FGFR3在生长骨骼中的复杂功能的许多特征被表征，这有助于识别治疗靶点，并推动治疗进展。2021 年 8 月，vosoritide 被批准用于治疗软骨发育不全，这是基于 C-利钠肽的稳定变体。随着几种概念上不同的 FGFR3 信号抑制剂在临床试验中取得进展，其他药物可能很快就会出现。在这里，我们回顾了当前的软骨发育不全疗法，描述了它们的机制，并阐明了导致它们发展的动机。我们还讨论了治疗软骨发育不全的观点，