Details
Stereochemistry | ACHIRAL |
Molecular Formula | C6H11O2.Na |
Molecular Weight | 138.1401 |
Optical Activity | NONE |
Defined Stereocenters | 0 / 0 |
E/Z Centers | 0 |
Charge | 0 |
SHOW SMILES / InChI
SMILES
[Na+].CCC(C)(C)C([O-])=O
InChI
InChIKey=OOPXYEYPPHJHSE-UHFFFAOYSA-M
InChI=1S/C6H12O2.Na/c1-4-6(2,3)5(7)8;/h4H2,1-3H3,(H,7,8);/q;+1/p-1
2,2-DIMETHYLBUTYRIC ACID (HQK-1001) is an orally administered SCFAD (Short Chain Fatty Acid Derivative), which has shown an excellent safety profile and biologic effects on fetal hemoglobin induction and red blood cell production in the laboratory, relevant animal models, and in clinical trials carried out in healthy human subjects as well as patients with sickle cell disease and beta thalassemia. The compound has received Orphan Drug Designation in the United States and Europe for both sickle cell disease and beta thalassemia. HemaQuest Pharmaceuticals was developing HQK-1001 for the oral treatment of sickle cell anaemia and beta thalassaemia. HQK-1001 has been evaluated in phase II trials for beta thalassaemia and sickle cell anaemia.
Originator
Approval Year
Sample Use Guides
In Vivo Use Guide
Sources: https://www.ncbi.nlm.nih.gov/pubmed/23828223
Hemoglobin SS or S/β(0) thalassemia: HQK-1001 was administered daily for 26 weeks at 30 mg/kg (n = 15), 40 mg/kg (n = 18) and 50 mg/kg (n = 19), either alone (n = 21) or with hydroxyurea (n = 31).
Route of Administration:
Oral
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FDA ORPHAN DRUG |
265108
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EU-Orphan Drug |
EU/3/09/621
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admin on Fri Dec 15 16:30:27 GMT 2023 , Edited by admin on Fri Dec 15 16:30:27 GMT 2023
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FDA ORPHAN DRUG |
259608
Created by
admin on Fri Dec 15 16:30:27 GMT 2023 , Edited by admin on Fri Dec 15 16:30:27 GMT 2023
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3934-02-9
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53247304
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100000178199
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SUB193821
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XKM99SPB5I
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admin on Fri Dec 15 16:30:27 GMT 2023 , Edited by admin on Fri Dec 15 16:30:27 GMT 2023
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ACTIVE MOIETY
SUBSTANCE RECORD