Sobetirome: The past, present and questions about the future

Jan Lammel Lindemann, Paul Webb

Research output: Contribution to journalReview articlepeer-review

22 Scopus citations


Sobetirome binds selectively to the main hepatic form of thyroid hormone (TH) receptor, TRβ1, compared to TR1, which is principally responsible for thyrotoxic effects on heart, muscle and bone. Sobetirome also preferentially accumulates in liver. It was originally envisaged that sobetirome could be used to stimulate hepatic pathways that lower cholesterol without harmful side effects and might be used in conjunction with statins. Indeed, sobetirome progressed through preclinical animal studies and Phase I human clinical trials with excellent results and without obvious harmful side effects. Despite the fact that cardiovascular disease remains a major cause of mortality and that new therapies are desperately needed, it is unlikely that sobetirome will progress in further human clinical trials in the near future. The emergence of alternative cholesterol-lowering therapeutics may render selective thyromimetics redundant. Further, fears of thyrotoxic effects in the heart and emergence of cartilage defects in dogs after long-term use of eprotirome, a similar though not identical compound, has reduced enthusiasm for this strategy. We argue that it is nevertheless important to explore uses of sobetirome in humans; more treatment strategies would help patients with hard-to-treat dyslipidemias. Sobetirome may also have additional applications in orphan indications and short-term controlled weight loss.

Original languageEnglish (US)
Pages (from-to)145-149
Number of pages5
JournalExpert Opinion on Therapeutic Targets
Issue number2
StatePublished - Feb 1 2016


  • Cholesterol lowering
  • Dyslipidemia
  • Eprotirome
  • GC-1
  • KB2115
  • Obesity
  • Sobetirome
  • Thyroid hormone
  • Thyroid hormone receptors
  • Thyromimetic

ASJC Scopus subject areas

  • Molecular Medicine
  • Pharmacology
  • Drug Discovery
  • Clinical Biochemistry


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