Sobetirome Explained

Routes Of Administration:	Oral
Legal Status:	Investigational
Synonyms:	GC-1
Cas Number:	211110-63-3
Chebi:	79988
Chembl:	107400
Chemspiderid:	8037944
Iuphar Ligand:	2639
Kegg:	D09381
Pubchem:	9862248
Unii:	XQ31741E9Q
Drugbank:	DB07425
Stdinchi:	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)
Stdinchikey:	QNAZTOHXCZPOSA-UHFFFAOYSA-N
Smiles:	CC1=CC(=CC(=C1CC2=CC(=C(C=C2)O)C(C)C)C)OCC(=O)O
Iupac Name:	2-[4-[(4-Hydroxy-3-propan-2-ylphenyl)methyl]-3,5-dimethylphenoxy]acetic acid
C:	20
H:	24
O:	4

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]

Notes and References

1. Lammel Lindemann . Jan . Webb . Paul . Sobetirome: the past, present and questions about the future . Expert Opinion on Therapeutic Targets . 2016 . 20 . 2 . 145–149 . 10.1517/14728222.2016.1090429 . 26565124 . 1744-7631. free .
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 . Promyelinating drugs promote functional recovery in an autism spectrum disorder mouse model of Pitt–Hopkins syndrome . Brain . 1 August 2023 . 146 . 8 . 3331–3346 . 10.1093/brain/awad057 . free . 37068912 . 10393406 .
3. Kosar . Karis . Cornuet . Pamela . Singh . Sucha . Liu . Silvia . Nejak-Bowen . Kari . The Thyromimetic Sobetirome (GC-1) Alters Bile Acid Metabolism in a Mouse Model of Hepatic Cholestasis . The American Journal of Pathology . May 2020 . 190 . 5 . 1006–1017 . 10.1016/j.ajpath.2020.01.015 . 32205094 . 7221298 . 0002-9440.
4. Book: Shah . Kamal . Chauhan . Durgesh Nandini . Chauhan . Nagendra Singh . Mishra . Pradeep . Recent Advancement in Prodrugs . 13 May 2020 . CRC Press . 978-1-000-05799-7 . en.
5. Matralis . Alexios . Karampitsakos . Theodoros . Papaioannou . Ourania . Katsaras . Matthaios . Sampsonas . Fotios . Bouros . Demosthenes . Aidinis . Vassilis . Tzouvelekis . Argyrios . Biosynthesis and implementation of Thyroid Receptor beta (TRß) agonists (thyromimetics) for the treatment of pulmonary fibrosis . European Respiratory Journal . 5 September 2021 . 58 . suppl 65 . OA4334 . 10.1183/13993003.congress-2021.OA4334 . 244680799 . en . 0903-1936.
6. Web site: Hartley . Pamela . $100,000 Gift Speeds Exploration of Sobetirome as COVID-19 Treatment . medicine.yale.edu . 15 September 2023 . en.
7. Web site: Search Orphan Drug Designations and Approvals . www.accessdata.fda.gov . 15 September 2023.