Fosdenopterin Hydrobromide — Active Pharmaceutical Ingredient

Fosdenopterin Hydrobromide (1 brand name, 1 dosage form, 1 route)

Available dosage forms: POWDER.

Routes of administration: INTRAVENOUS.

Brand Names

• NULIBRY

Indications and Usage

NULIBRY is indicated to reduce the risk of mortality in patients with molybdenum cofactor deficiency (MoCD) Type A.

Contraindications

None.

Mechanism of Action

Patients with MoCD Type A have mutations in the MOCS1 gene leading to deficient MOCS1A/B dependent synthesis of the intermediate substrate, cPMP. Substrate replacement therapy with NULIBRY 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 sulfite oxidase (SOX), an enzyme that reduces levels of neurotoxic sulfites.