Sepiapterin

ALIMENTARY TRACT AND METABOLISM

Key facts

Sepiapterin is an onp drug catalogued in DrugCentral. It was first approved by the EMA in 2025. DrugCentral lists 1 molecular target, including PAH. Recorded indications include Hyperphenylalaninemia (HPA).

• Approval status: onp (first approved 2025 by the EMA).
• Molecular targets: PAH (1 listed in DrugCentral).
• Indications: Hyperphenylalaninemia (HPA).
• ATC code: A16AX28 (Various alimentary tract and metabolism products).
• Molecular formula: C9H11N5O3; molecular weight 237.22 g/mol.

Indications & contraindications

Drug–disease relationships curated by DrugCentral (OMOP / SNOMED concepts).

Indications (1)

• Hyperphenylalaninemia (HPA)

Mechanism & targets

Molecular targets (1)

Target	Gene	Action	Activity
Phenylalanine-4-hydroxylase	PAH	activator	—

Regulatory approvals (3)

Date	Agency	Marketed as	Applicant	Orphan
2025-12-22	PMDA	—	PTC Therapeutics K.K.	Yes
2025-07-28	FDA	—	PTC Therapeutics	Yes
2025-06-19	EMA	—	PTC Therapeutics International Limited	Yes

ATC classification

ATC code	Therapeutic hierarchy
A16AX28	ALIMENTARY TRACT AND METABOLISM › OTHER ALIMENTARY TRACT AND METABOLISM PRODUCTS › OTHER ALIMENTARY TRACT AND METABOLISM PRODUCTS › Various alimentary tract and metabolism products

Pharmacogenomics

Drug-label and guideline annotations from PharmGKB and CPIC.

• EMA label: ABCG2
• FDA label: ABCG2 · No Clinical PGx
• HCSC label: ABCG2

Chemical structure

Full physicochemical, ADMET and 3D-structure analysis of Sepiapterin is on its molecule page.

• Molecular formula: C₉H₁₁N₅O₃
• Molecular weight: 237.22 g/mol
• CAS number: —
• InChIKey: VPVOXUSPXFPWBN-VKHMYHEASA-N
• PubChem CID: 135398579

C[C@@H](C(=O)C1=NC2=C(NC1)N=C(NC2=O)N)O

Frequently asked questions

Answers are generated from the structured data on this page, not written by hand.

What is Sepiapterin used for?

DrugCentral lists the following indications for Sepiapterin: Hyperphenylalaninemia (HPA).

How does Sepiapterin work?

Its molecular targets in DrugCentral include PAH (activator).

When was Sepiapterin first approved?

Sepiapterin was first approved by the EMA in 2025.

What is the chemical formula of Sepiapterin?

Sepiapterin has the molecular formula C9H11N5O3 and a molecular weight of 237.22 g/mol.

Sources

• DrugCentral drug card 5801 — structure, indications, targets, approvals, pharmacologic classes (CC BY-SA 4.0)
• ChEMBL CHEMBL1255653 — compound record and bioactivity
• PubChem CID 135398579 — chemical identifiers
• DrugBank DB16326 — drug record
• FDA UNII CJQ26KO7HP — substance identity
• DailyMed: FDA labels for Sepiapterin — current prescribing information (label search)
• UniProt P00439 (PAH) — target protein
• PharmGKB / CPIC — pharmacogenomic label annotations

How this page is built: methodology · editorial policy & corrections.