Chemical formula: C₁₀H₁₄N₅O₈P Molecular mass: 363.223 g/mol
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.
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 | |
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 |
Fosdenopterin is an active ingredient of these brands:
United States (US)
Austria (AT)
Croatia (HR)
Israel (IL)
Lithuania (LT)
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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