Sobetirome

Sobetirome (GC-1) is a thyromimetic drug that binds to the thyroid hormone receptor TRβ1 preferentially compared to TRα1. It has been investigated for the treatment of dyslipidemia, obesity,[1] Pitt–Hopkins syndrome,[2] cholestatic liver disease,[3] multiple sclerosis,[4] bleomycin-induced lung fibrosis,[5] and COVID-19 caused ARDS.[6] It was designated as an orphan drug by the FDA for the treatment of X-linked adrenoleukodystrophy.[7]

Sobetirome
Clinical data
Other namesGC-1
Legal status
Legal status
  • Investigational
Identifiers
  • 2-[4-[(4-Hydroxy-3-propan-2-ylphenyl)methyl]-3,5-dimethylphenoxy]acetic acid
CAS Number
PubChem CID
IUPHAR/BPS
DrugBank
ChemSpider
UNII
KEGG
ChEBI
ChEMBL
CompTox Dashboard (EPA)
Chemical and physical data
FormulaC20H24O4
Molar mass328.408 g·mol−1
3D model (JSmol)
  • CC1=CC(=CC(=C1CC2=CC(=C(C=C2)O)C(C)C)C)OCC(=O)O
  • InChI=1S/C20H24O4/c1-12(2)17-9-15(5-6-19(17)21)10-18-13(3)7-16(8-14(18)4)24-11-20(22)23/h5-9,12,21H,10-11H2,1-4H3,(H,22,23)
  • Key:QNAZTOHXCZPOSA-UHFFFAOYSA-N

References
  1. Lammel Lindemann, Jan; Webb, Paul (2016). "Sobetirome: the past, present and questions about the future". Expert Opinion on Therapeutic Targets. 20 (2): 145–149. doi:10.1517/14728222.2016.1090429. ISSN 1744-7631. PMID 26565124.
  2. Bohlen, Joseph F; Cleary, Colin M; Das, Debamitra; Sripathy, Srinidhi Rao; Sadowski, Norah; Shim, Gina; Kenney, Rakaia F; Buchler, Ingrid P; Banerji, Tapasree; Scanlan, Thomas S; Mulkey, Daniel K; Maher, Brady J (1 August 2023). "Promyelinating drugs promote functional recovery in an autism spectrum disorder mouse model of Pitt–Hopkins syndrome". Brain. 146 (8): 3331–3346. doi:10.1093/brain/awad057.
  3. Kosar, Karis; Cornuet, Pamela; Singh, Sucha; Liu, Silvia; Nejak-Bowen, Kari (May 2020). "The Thyromimetic Sobetirome (GC-1) Alters Bile Acid Metabolism in a Mouse Model of Hepatic Cholestasis". The American Journal of Pathology. 190 (5): 1006–1017. doi:10.1016/j.ajpath.2020.01.015. ISSN 0002-9440. PMC 7221298. PMID 32205094.
  4. Shah, Kamal; Chauhan, Durgesh Nandini; Chauhan, Nagendra Singh; Mishra, Pradeep (13 May 2020). Recent Advancement in Prodrugs. CRC Press. ISBN 978-1-000-05799-7.
  5. Matralis, Alexios; Karampitsakos, Theodoros; Papaioannou, Ourania; Katsaras, Matthaios; Sampsonas, Fotios; Bouros, Demosthenes; Aidinis, Vassilis; Tzouvelekis, Argyrios (5 September 2021). "Biosynthesis and implementation of Thyroid Receptor beta (TRß) agonists (thyromimetics) for the treatment of pulmonary fibrosis". European Respiratory Journal. 58 (suppl 65): OA4334. doi:10.1183/13993003.congress-2021.OA4334. ISSN 0903-1936.
  6. Hartley, Pamela. "$100,000 Gift Speeds Exploration of Sobetirome as COVID-19 Treatment". medicine.yale.edu. Retrieved 15 September 2023.
  7. "Search Orphan Drug Designations and Approvals". www.accessdata.fda.gov. Retrieved 15 September 2023.
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