Fosdenopterin

Chemical formula: C₁₀H₁₄N₅O₈P  Molecular mass: 363.223 g/mol 

Active ingredient description

Patients with MoCD Type A have mutations in the Molybdenum Cofactor Synthesis 1 (MOCS1) gene leading to deficient MOCS1A/B dependent synthesis of the intermediate substrate, cPMP. Substrate replacement therapy with fosdenopterin provides an exogenous source of cPMP, which is converted to molybdopterin. Molybdopterin is then converted to molybdenum cofactor, which is needed for the activation of molybdenum-dependent enzymes, including sulphite oxidase (SOX), an enzyme that reduces levels of neurotoxic sulphites.

Medicine classification

This medicinal substance has been classified in the anatomical therapeutic chemical (ATC) classification according to its main therapeutic use as follows:

ATC code Group title Classification
A16AX19 A Alimentary tract and metabolism → A16 Other alimentary tract and metabolism products → A16A Other alimentary tract and metabolism products → A16AX Various alimentary tract and metabolism products
Discover more medicines within A16AX19

Product monographs

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

Title Information Source Document Type  
NULIBRY Powder for solution for injection European Medicines Agency (EU) MPI, EU: SmPC

Structural formula

Graphic representation of the active ingredient's molecular structure

External identifiers

CAS Substance: 150829-29-1
DrugBank Drug: DB16628
KEGG Drug: D11779
RxNorm Ingredient: 2531288
SNOMED-CT Concept: 1149378000
Fosdenopterin (substance)
UNII Identifier: 4X7K2681Y7
FOSDENOPTERIN

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