Obefazimod: A First-in-class Drug for the Treatment of Ulcerative Colitis

Séverine Vermeire, Virginia Solitano, Laurent Peyrin-Biroulet, Herbert Tilg, Silvio Danese, Hartmut Ehrlich, Didier Scherrer, Paul Gineste, Laurence D'agay, Bruce E. Sands

Research output: Contribution to journalReview articlepeer-review

2 Scopus citations


Biologic agents and oral small molecules are the mainstays of inflammatory bowel disease [IBD] management. However, an unmet clinical need remains for additional agents with novel mechanism of action which are effective, safe, and disease-modifying; this is due to the substantial proportion of patients who do not respond, lose response, or develop intolerance to currently marketed products. microRNAs [miRNAs] that play a role in the modulation of signal transduction pathways implicated in the development of IBD hold the potential to be used as therapeutic targets. Recently, a novel first-in-class compound, obefazimod, originally conceived as a human immunodeficiency virus [HIV] infection drug, has shown great promise in phase II induction trials for ulcerative colitis [UC] patients. Findings from the maintenance phases of trials showed that long-term obefazimod treatment provides continued improvement in clinical symptoms of disease, with a substantial proportion of patients in clinical remission, and an overall good safety profile. With a novel mechanism of action, obefazimod is an orally available small molecule with anti-inflammatory properties through the specific and selective upregulation of miR-124 expression. The aim of this paper is to critically review the available evidence related to pharmacokinetics and pharmacodynamics, and to discuss the potential clinical implications of this first-in-class oral small molecule.

Original languageEnglish
Pages (from-to)1689-1697
Number of pages9
JournalJournal of Crohn's and Colitis
Issue number10
StatePublished - 1 Oct 2023


  • ABX464
  • Oral small molecules
  • miRNAs
  • obefazimod
  • ulcerative colitis


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