Indole therapeutics: Latest FDA updates, ongoing trials, and future directions.
Noor-Ul-Huda Butt, Sultan Nacak Baytas
Drug discovery today October 1, 2025 DOI: 10.1016/j.drudis.2025.104471 via PubMed
Summary
Indole-based compounds are versatile molecules used in drugs for cancer, viral infections, neurological disorders, and other diseases. This review systematically examines FDA-approved, withdrawn, and investigational indole-containing drugs from the past decade, focusing on clinical outcomes, regulatory decisions, and therapeutic repositioning. It describes a shift from cytotoxic to targeted anticancer agents, the rise of indole-based antivirals during the COVID-19 pandemic, and growing interest in neuroactive indoles, including psychedelic compounds. The review also notes the underrepresentation of indole-based antibiotics and outlines progress in epigenetic and metabolic modulators, providing a framework for future drug development.
Study at a glance
| Characteristics | Review Peer reviewed |
|---|---|
| Keywords | FDA Approvals Cancer Clinical trials Drug development Indole |
| Citations | 4 |
| Key finding | Indole-based compounds have evolved from cytotoxic to targeted anticancer agents, gained prominence as antivirals during the COVID-19 pandemic, and are increasingly explored as neuroactive agents, though indole-based antibiotics remain underrepresented. |
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 This review systematically analyses FDA-approved, withdrawn, and investigational indole-containing drugs over the past decade, focusing on evolving clinical outcomes, regulatory decisions, and therapeutic repositioning.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.