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

Butt N., BAYTAŞ S.

Drug Discovery Today, vol.30, no.10, 2025 (SCI-Expanded) identifier identifier

  • Publication Type: Article / Review
  • Volume: 30 Issue: 10
  • Publication Date: 2025
  • Doi Number: 10.1016/j.drudis.2025.104471
  • Journal Name: Drug Discovery Today
  • Journal Indexes: Science Citation Index Expanded (SCI-EXPANDED), Scopus, Academic Search Premier, Aerospace Database, Biotechnology Research Abstracts, Communication Abstracts, EMBASE, International Pharmaceutical Abstracts, MEDLINE, Metadex, Civil Engineering Abstracts
  • Keywords: cancer, clinical trials, drug development, FDA approvals, indole, neurological disorders, viral infectious
  • Gazi University Affiliated: Yes

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 This review systematically analyses FDA-approved, withdrawn, and investigational indole-containing drugs over the past decade, focusing on evolving clinical outcomes, regulatory decisions, and therapeutic repositioning.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.