Sepiapterin
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| IUPAC name
2-amino-6-[(2S)-2-hydroxypropanoyl]-7,8-dihydro-1H-pteridin-4-one
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| Other names
Sephience
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| Identifiers | |
3D model (JSmol)
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| ChEMBL | |
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PubChem CID
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| Properties | |
| C9H11N5O3 | |
| Molar mass | 237.22 g/mol |
| Pharmacology | |
| None | |
| By mouth | |
| Legal status | |
Except where otherwise noted, data are given for materials in their standard state (at 25 °C [77 °F], 100 kPa).
Infobox references
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Sepiapterin, sold under the brand name Sephience, is a medication used for the treatment of hyperphenylalaninemia.[1][2]
The most common side effects are upper respiratory tract infection, headache, diarrhea, abdominal pain, hyperphenylalaninemia and discoloration of feces.[1]
Medical uses
Sepiapterin is indicated for the treatment of hyperphenylalaninemia in people with phenylketonuria.[1][2]
Side effects
The most common side effects are upper respiratory tract infection, headache, diarrhea, abdominal pain, hyperphenylalaninemia and discoloration of feces.[1]
Chemistry
Sepiapterin is known as 2-amino-6-[(2S)-2-hydroxypropanoyl]-7,8-dihydro-1H-pteridin-4-one and 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.
Society and culture
Legal status
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.[1] The applicant for this medicinal product is PTC Therapeutics International Limited.[1] Sepiapterin was authorized for medical use in the European Union in June 2025.[1][2]
Research
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.[3]
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.[4]
References
- ^ a b c d e f g h "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.
- ^ a b c d "Sephience Product information". Union Register of medicinal products. 25 June 2025. Retrieved 27 June 2025.
- ^ 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.