Sepiapterin
 | |
| Names | |
|---|---|
| IUPAC name
2-amino-6-[(2S)-2-hydroxypropanoyl]-7,8-dihydro-1H-pteridin-4-one
| |
| Identifiers | |
3D model (JSmol)
|
|
| ChEMBL | |
| ChemSpider | |
| KEGG | |
PubChem CID
|
|
| UNII | |
| |
| |
| Properties | |
| C9H11N5O3 | |
| Molar mass | 237.22 g/mol |
| Pharmacology | |
| None | |
Except where otherwise noted, data are given for materials in their standard state (at 25 °C [77 °F], 100 kPa).
| |
Sepiapterin, also known as 2-amino-6-[(2S)-2-hydroxypropanoyl]-7,8-dihydro-1H-pteridin-4-one, is a member of the pteridine class of organic chemicals.
Sepiapterin can be metabolized into tetrahydrobiopterin via a salvage pathway. Tetrahydrobiopterin is an essential cofactor in humans for breakdown of phenylalanine and a catalyst of the metabolism of phenylalanine, tyrosine, and tryptophan to precursors of the neurotransmitters dopamine and serotonin.
Deficiency of tetrahydrobiopterin can cause toxic buildup of phenylalanine (phenylketonuria) as well as deficiencies of dopamine, norepinephrine, and epinephrine, leading to dystonia and other neurological illnesses. This has led to clinical study of sepiapterin in humans to treat tetrahydrobiopterin deficiency.[1]
Since atherosclerosis and other circulatory diseases associated with diabetes are also associated with tetrahydrobiopterin deficiency, animal studies of the value of sepiaterin in these vascular diseases have been done. These studies show that relaxation of the blood vessels studied was impaired after animals were given sepiapterin, even though their levels of tetrahydrobiopterin were replenished.[2]
Society and culture
[edit]Legal status
[edit]In April 2025, the Committee for Medicinal Products for Human Use of the European Medicines Agency adopted a positive opinion, recommending the granting of a marketing authorization for the medicinal product Sephience, intended for the treatment of hyperphenylalaninemia in adults and children with phenylketonuria.[3] The applicant for this medicinal product is PTC Therapeutics International Limited.[3]
References
[edit]- ^ Smith N, Longo N, Levert K, Hyland K, Blau N (1 April 2019). "Phase I clinical evaluation of CNSA-001 (sepiapterin), a novel pharmacological treatment for phenylketonuria and tetrahydrobiopterin deficiencies, in healthy volunteers". Molecular Genetics and Metabolism. 126 (4): 406–412. doi:10.1016/j.ymgme.2019.02.001. ISSN 1096-7192. PMID 30922814. S2CID 85564348.
- ^ Vasquez-Vivar J, Duquiane D, Whitsett J, Kalyanaraman B, Rajagopalan S (1 October 2002). "Altered Tetrahydrobiopterin Metabolism in Atherosclerosis". Arteriosclerosis, Thrombosis, and Vascular Biology. 22 (10): 1655–1661. doi:10.1161/01.ATV.0000029122.79665.D9. PMID 12377745.
- ^ a b "Sephience EPAR". European Medicines Agency (EMA). 25 April 2025. Retrieved 2 May 2025. Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
 | This article about a ketone is a stub. You can help Wikipedia by expanding it. |