Doxecitine
CAS951-77-9
MF C9H13N3O4
11/3/2025, FDA 2025, To treat thymidine kinase 2 deficiency in patients who start to show symptoms when they are 12 years old or younger
- CYTIDINE, 2′-DEOXY-
- dCYD
- DEOXYCYTIDINE
4-amino-1-[(2R,4S,5R)-4-hydroxy-5-(hydroxymethyl)oxolan-2-yl]-1,2-dihydropyrimidin-2-one
Doxecitine is a pyrimidine nucleoside used to treat thymidine kinase 2 deficiency.
Doxecitine is a synthetic form of the naturally occurring pyrimidine deoxyribonucleoside deoxycytidine. It is an essential component of the deoxyribonucleotide pool required for DNA synthesis and repair. Doxecitine is currently approved and marketed as a fixed-dose combination therapy with thymidine (KYGEVVI). This combination is the first and only approved treatment for Thymidine Kinase 2 deficiency.5,6
Deoxycytidine is a deoxyribonucleoside, a component of deoxyribonucleic acid. It is similar to the ribonucleoside cytidine, but with one hydroxyl group removed from the C2′ position. Deoxycytidine can be phosphorylated at C5′ of the deoxyribose by deoxycytidine kinase, converting it to deoxycytidine monophosphate (dCMP), a DNA precursor.[1] dCMP can be converted to dUMP and dTMP.
Doxecitine is the international nonproprietary name.[2]
SYN
Graham A. Mock, Douglas H. Lovern, “N.sup.4 -substituted 2′-deoxycytidine compounds, oligonucleotides including N.sup.4 -labeled 2′-deoxycytidines, and a process for making oligonucleotides with N-modified 2′-deoxycytidines.” U.S. Patent US5633364, issued April, 1995.US5633364
PAT
https://patentscope.wipo.int/search/en/detail.jsf?docId=US37089691&_cid=P21-MJXONM-10154-1
PAT
https://patentscope.wipo.int/search/en/detail.jsf?docId=WO1982003079&_cid=P21-MJXONM-10154-1
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- Chow E, Miller L, Clearman A, Arnold P, Koenig MK, Russo SN: Doxecitine and doxribtimine treatment in an adult patient with thymidine kinase 2 deficiency. Mol Genet Metab. 2025 Aug;145(4):109159. doi: 10.1016/j.ymgme.2025.109159. Epub 2025 Jun 3. [Article]
- Mittur A, VanMeter SA, Orujov E, Glidden P: Pharmacokinetics and Safety of a 1:1 Mixture of Doxecitine and Doxribtimine: Open-label Phase 1 Single Ascending Dose and Food Effect Studies in Healthy Adults. Clin Ther. 2024 Jul;46(7):576-587. doi: 10.1016/j.clinthera.2024.06.006. Epub 2024 Jul 18. [Article]
- Lopez-Gomez C, Levy RJ, Sanchez-Quintero MJ, Juanola-Falgarona M, Barca E, Garcia-Diaz B, Tadesse S, Garone C, Hirano M: Deoxycytidine and Deoxythymidine Treatment for Thymidine Kinase 2 Deficiency. Ann Neurol. 2017 May;81(5):641-652. doi: 10.1002/ana.24922. Epub 2017 May 4. [Article]
- FDA Approved Drug Products: KYGEVVI (doxecitine and doxribtimine) powder, for oral solution (November 2025) [Link]
- UCB: New data on investigational therapy for thymidine kinase 2 deficiency presented at Muscular Dystrophy Association (MDA) 2025 Conference [Link]
- PR Newswire: U.S. FDA approves KYGEVVI™ (doxecitine and doxribtimine), the first and only treatment for adults and children living with thymidine kinase 2 deficiency (TK2d) [Link]
| Names | |
|---|---|
| IUPAC name2′-deoxycytidine | |
| Systematic IUPAC name4-Amino-1-[(2R,4S,5R)-4-hydroxy-5-(hydroxymethyl)oxolan-2-yl]pyrimidin-2(1H)-one | |
| Other namesdoxecitine | |
| Identifiers | |
| CAS Number | 951-77-9 |
| 3D model (JSmol) | Interactive image |
| ChEBI | CHEBI:15698 |
| ChEMBL | ChEMBL66115 |
| ChemSpider | 13117 |
| ECHA InfoCard | 100.012.231 |
| MeSH | Deoxycytidine |
| PubChem CID | 13711 |
| UNII | 0W860991D6 |
| CompTox Dashboard (EPA) | DTXSID70883620 |
| InChI | |
| SMILES | |
| Properties | |
| Chemical formula | C9H13N3O4 |
| Molar mass | 227.217 |
| Except where otherwise noted, data are given for materials in their standard state (at 25 °C [77 °F], 100 kPa).Infobox references | |
References
- Staub M, Eriksson S (2006). “The Role of Deoxycytidine Kinase in DNA Synthesis and Nucleoside Analog Activation”. In Peters GJ (ed.). Deoxynucleoside Analogs In Cancer Therapy. Cancer Drug Discovery and Development. Humana Press. pp. 29–52. doi:10.1007/978-1-59745-148-2_2. ISBN 978-1-59745-148-2.
- World Health Organization (2022). “International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 87”. WHO Drug Information. 36 (1). hdl:10665/352794.
- Kim KW, Roh JK, Wee HJ, Kim C (2016). “Molecular Targeted Anticancer Drugs”. In Kim KW, Roh JK, Wee HJ, Kim C (eds.). Cancer Drug Discovery: Science and History. Springer Netherlands. pp. 175–238. doi:10.1007/978-94-024-0844-7_9. ISBN 978-94-024-0844-7.
- Guo M, Zhang L, Du Y, Du W, Liu D, Guo C, et al. (March 2018). “Enrichment and Quantitative Determination of 5-(Hydroxymethyl)-2′-deoxycytidine, 5-(Formyl)-2′-deoxycytidine, and 5-(Carboxyl)-2′-deoxycytidine in Human Urine of Breast Cancer Patients by Magnetic Hyper-Cross-Linked Microporous Polymers Based on Polyionic Liquid”. Analytical Chemistry. 90 (6): 3906–3913. doi:10.1021/acs.analchem.7b04755. PMID 29316399.
- “FDA approves 1st drug for thymidine kinase 2 deficiency”. U.S. Food and Drug Administration. 3 November 2025. Retrieved 4 November 2025.
This article incorporates text from this source, which is in the public domain.
External links
- “Doxecitine ( Code – C420 )”. EVS Explore.
- MeSH 68003841
/////////doxecitine, deoxycytidine, CYTIDINE, 2′-DEOXY-, dCYD, FDA 2025, APPROVALS 2025