Indole therapeutics: Latest FDA updates, ongoing trials, and future directions

Abstract

Indole-based compounds serve as versatile pharmacophores across anticancer, antiviral, neurological, antimicrobial, and metabolic therapies. This review systematically analyses FDA-approved, withdrawn, and investigational indole-containing drugs over the past decade, focusing on evolving clinical outcomes, regulatory decisions, and therapeutic repositioning. It explores the shift from cytotoxic to targeted anticancer agents, the rise of indole-based antivirals in response to the COVID-19 pandemic, and the expanding interest in neuroactive indoles, particularly psychedelic compounds. Additionally, it highlights the underrepresentation of indole-based antibiotics and outlines progress in epigenetic and metabolic modulators. Through a detailed evaluation of pharmacological classes, clinical trial data, and structural characteristics, this review presents a comprehensive framework to guide future optimization and multi-target development of indole scaffolds.