Indole therapeutics: Latest FDA updates, ongoing trials, and future directions.
Drug discovery today October 1, 2025 Noor-Ul-Huda Butt, Sultan Nacak Baytas 4 citations
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 This review systematically examines FDA-approved, withdrawn, and investigational indole-containing drugs from the past decade, focusing on clinical outcomes, regulatory decisions, and therapeutic repositioning.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.