Sepiapterin

Chemical formula: C₉H₁₁N₅O₃  Molecular mass: 237.086 g/mol  PubChem compound: 135398579

Active ingredient description

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.

Product monographs

Competent medicine agencies globally have authorized commercialization of this active ingredient according to these medication package inserts (MPIs):

Document Type Information Source  
 SEPHIENCE Oral powder MPI, EU: SmPC European Medicines Agency (EU)

Structural formula

Graphic representation of the active ingredient's molecular structure

External identifiers

DrugBank Drug: DB16326
KEGG Drug: D12286
PubChem Compound: 135398579
UNII Identifier: CJQ26KO7HP
SEPIAPTERIN

Medicines

Sepiapterin is an active ingredient of these brands:

Austria (AT)

Lithuania (LT)

United States (US)

Note the following: The list of brand names is continuously updated, and thus does not include the total of products circulating worldwide.

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