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sepiapterin 🐶 Veterinary Use | Indications/Contra | FAERs-F | FAERs-M | Orange Bk | PK/Tox | Related Drugs | BioActivity |
| Stem definition | Drug id | CAS RN |
|---|---|---|
| pteridine derivatives | 5801 | 17094-01-08 |
Description:
| Molecule | Description |
|---|---|
|
Synonyms:
|
Sepiapterin is a natural precursor of the enzymatic co-factor BH4, a critical co-factor for phenylalanine hydroxylase (PAH). Sepiapterin acts as a dual pharmacological chaperone (sepiapterin and BH4 each with its own binding affinity to variant PAH), including PAH variants commonly found in PKU and known to be insensitive to BH4, to improve the activity of the defective PAH enzyme, achieving a high concentration of BH4 intracellularly. By enhancing the conformational stability of misfolded PAH enzyme and increasing the intracellular concentrations of BH4, sepiapterin is able to effectively reduce blood Phe levels.
Legend: OFP - off patent OFM - off market ONP - on patent |
Drug dosage:
None
ADMET properties:
None
Predicted pharmacokinetic/toxicological properties (PK-AZ):
None
Approvals:
| Date | Agency | Company | Orphan |
|---|---|---|---|
| June 19, 2025 | EMA | PTC Therapeutics International Limited | |
| July 28, 2025 | FDA | PTC Therapeutics | |
| Dec. 22, 2025 | PMDA | PTC Therapeutics K.K. |
FDA Adverse Event Reporting System (Female)
None
FDA Adverse Event Reporting System (Male)
None
FDA Adverse Event Reporting System (Geriatric)
None
FDA Adverse Event Reporting System (Pediatric)
None
Pharmacologic Action:
| Source | Code | Description |
|---|---|---|
| ATC | A16AX28 | ALIMENTARY TRACT AND METABOLISM OTHER ALIMENTARY TRACT AND METABOLISM PRODUCTS OTHER ALIMENTARY TRACT AND METABOLISM PRODUCTS Various alimentary tract and metabolism products |
| MeSH PA | D020536 | Enzyme Activators |
Related Drugs by ATC class:
A16AX Various alimentary tract and metabolism products 23 drugs
Drug Use | Suggest Off label Use Form| |View source of the data|
| Disease | Relation | SNOMED_ID | DOID |
|---|---|---|---|
| Hyperphenylalaninemia (HPA) | indication | 68528007 |
🐶 Veterinary Drug Use
None
🐶 Veterinary products
None
Acid dissociation constants calculated using MoKa v3.0.0
None
Orange Book patent data (new drug applications)
| Formulation strength | Trade name | Applicant | Application number | Approval date | Type | Dose form | Route | Patent number | Patent expiration date | Patent use |
|---|---|---|---|---|---|---|---|---|---|---|
| 1GM/PACKET | SEPHIENCE | PTC THERAP | N219666 | July 28, 2025 | RX | POWDER | ORAL | 11072614 | April 16, 2038 | TREATMENT OF HYPERPHENYLALANINEMIA (HPA) IN ADULT AND PEDIATRIC PATIENTS WITH SEPIAPTERIN-RESPONSIVE PHENYLKETONURIA (PKU) BY ADMINISTERING SEPIAPTERIN |
| 250MG/PACKET | SEPHIENCE | PTC THERAP | N219666 | July 28, 2025 | RX | POWDER | ORAL | 11072614 | April 16, 2038 | TREATMENT OF HYPERPHENYLALANINEMIA (HPA) IN ADULT AND PEDIATRIC PATIENTS WITH SEPIAPTERIN-RESPONSIVE PHENYLKETONURIA (PKU) BY ADMINISTERING SEPIAPTERIN |
| 1GM/PACKET | SEPHIENCE | PTC THERAP | N219666 | July 28, 2025 | RX | POWDER | ORAL | 11752154 | Sept. 4, 2038 | TREATMENT OF HYPERPHENYLALANINEMIA (HPA) IN ADULT AND PEDIATRIC PATIENTS WITH SEPIAPTERIN-RESPONSIVE PHENYLKETONURIA (PKU) BY ADMINISTERING SEPIAPTERIN |
| 1GM/PACKET | SEPHIENCE | PTC THERAP | N219666 | July 28, 2025 | RX | POWDER | ORAL | 12213982 | Sept. 4, 2038 | TREATMENT OF HYPERPHENYLALANINEMIA (HPA) IN ADULT AND PEDIATRIC PATIENTS WITH SEPIAPTERIN-RESPONSIVE PHENYLKETONURIA (PKU) BY ADMINISTERING SEPIAPTERIN |
| 250MG/PACKET | SEPHIENCE | PTC THERAP | N219666 | July 28, 2025 | RX | POWDER | ORAL | 11752154 | Sept. 4, 2038 | TREATMENT OF HYPERPHENYLALANINEMIA (HPA) IN ADULT AND PEDIATRIC PATIENTS WITH SEPIAPTERIN-RESPONSIVE PHENYLKETONURIA (PKU) BY ADMINISTERING SEPIAPTERIN |
| 250MG/PACKET | SEPHIENCE | PTC THERAP | N219666 | July 28, 2025 | RX | POWDER | ORAL | 12213982 | Sept. 4, 2038 | TREATMENT OF HYPERPHENYLALANINEMIA (HPA) IN ADULT AND PEDIATRIC PATIENTS WITH SEPIAPTERIN-RESPONSIVE PHENYLKETONURIA (PKU) BY ADMINISTERING SEPIAPTERIN |
| 1GM/PACKET | SEPHIENCE | PTC THERAP | N219666 | July 28, 2025 | RX | POWDER | ORAL | 12257252 | March 6, 2042 | METHOD OF TREATING HYPERPHENYLALANINEMIA (HPA) IN ADULT AND PEDIATRIC PATIENTS WITH SEPIAPTERIN-RESPONSIVE PHENYLKETONURIA (PKU) BY ADMINISTERING SEPIAPTERIN WITH FOOD |
| 250MG/PACKET | SEPHIENCE | PTC THERAP | N219666 | July 28, 2025 | RX | POWDER | ORAL | 12257252 | March 6, 2042 | METHOD OF TREATING HYPERPHENYLALANINEMIA (HPA) IN ADULT AND PEDIATRIC PATIENTS WITH SEPIAPTERIN-RESPONSIVE PHENYLKETONURIA (PKU) BY ADMINISTERING SEPIAPTERIN WITH FOOD |
Orange Book exclusivity data (new drug applications)
| Formulation strength | Trade name | Applicant | Application number | Approval date | Type | Dose form | Route | Exclusivity date | Description |
|---|---|---|---|---|---|---|---|---|---|
| 1GM/PACKET | SEPHIENCE | PTC THERAP | N219666 | July 28, 2025 | RX | POWDER | ORAL | July 28, 2030 | NEW CHEMICAL ENTITY |
| 250MG/PACKET | SEPHIENCE | PTC THERAP | N219666 | July 28, 2025 | RX | POWDER | ORAL | July 28, 2030 | NEW CHEMICAL ENTITY |
| 1GM/PACKET | SEPHIENCE | PTC THERAP | N219666 | July 28, 2025 | RX | POWDER | ORAL | July 28, 2032 | TREATMENT OF HYPERPHENYLALANINEMIA (HPA) IN ADULT AND PEDIATRIC PATIENTS 1 MONTH OF AGE AND OLDER WITH SEPIAPTERIN-RESPONSIVE PHENYLKETONURIA (PKU) (TO BE USED IN CONJUNCTION WITH A PHENYLALANINE (PHE) - RESTRICTED DIET) |
| 250MG/PACKET | SEPHIENCE | PTC THERAP | N219666 | July 28, 2025 | RX | POWDER | ORAL | July 28, 2032 | TREATMENT OF HYPERPHENYLALANINEMIA (HPA) IN ADULT AND PEDIATRIC PATIENTS 1 MONTH OF AGE AND OLDER WITH SEPIAPTERIN-RESPONSIVE PHENYLKETONURIA (PKU) (TO BE USED IN CONJUNCTION WITH A PHENYLALANINE (PHE) - RESTRICTED DIET) |
Bioactivity Summary:
| Target | Class | Pharos | UniProt | Action | Type | Activity value (-log[M]) | Mechanism action | Bioact source | MoA source |
|---|---|---|---|---|---|---|---|---|---|
| Phenylalanine-4-hydroxylase | Enzyme | ACTIVATOR | UNKNOWN | DRUG LABEL |
External reference:
| ID | Source |
|---|---|
| 17094-01-8 | SECONDARY_CAS_RN |
| CHEMBL1255653 | ChEMBL_ID |
| C016727 | MESH_SUPPLEMENTAL_RECORD_UI |
| DB16326 | DRUGBANK_ID |
| 4044149 | VANDF |
| C0074349 | UMLSCUI |
| CHEBI:16095 | CHEBI |
| 11848 | INN_ID |
| 135398579 | PUBCHEM_CID |
| 396500 | MMSL |
| 44277 | MMSL |
| d10295 | MMSL |
| 020175 | NDDF |
| 14278 | IUPHAR_LIGAND_ID |
| CJQ26KO7HP | UNII |
| 2723056 | RXNORM |
| D12286 | KEGG_DRUG |
Pharmaceutical products:
| Product | Category | Ingredients | NDC | Form | Quantity | Route | Marketing | Label |
|---|---|---|---|---|---|---|---|---|
| Sephience | HUMAN PRESCRIPTION DRUG LABEL | 1 | 52856-201 | POWDER | 250 mg | ORAL | NDA | 28 sections |
| Sephience | HUMAN PRESCRIPTION DRUG LABEL | 1 | 52856-301 | POWDER | 1000 mg | ORAL | NDA | 28 sections |
| Sephience | HUMAN PRESCRIPTION DRUG LABEL | 1 | 60468-006 | POWDER | 1000 mg | ORAL | NDA | 28 sections |
| Sephience | HUMAN PRESCRIPTION DRUG LABEL | 1 | 60468-007 | POWDER | 250 mg | ORAL | NDA | 28 sections |