Daily Medication Pearl: Fosdenopterin (Nulibry)


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

Medication Pearl of the Day: Fosdenopterin (Nulibry)

Indication: Fosdenopterin(Nulibry) is cyclic pyranopterin monophosphate (cPMP) indicated to reduce the risk of mortality in patients with molybdenum cofactor deficiency (MoCD) Type A.


  • Dosing: Dosage may start from 0.4 mg/kg once daily to 0.9 mg/kg once daily.
  • Dosage forms: For injection 9.5 mg of fosdenopterin as a lyophilized powder or cake in a single-dose vial for reconstitution.
  • Adverse events:The most common adverse reactions (>25%) were catheter-related complications, pyrexia, viral infection, pneumonia, otitis media, vomiting, cough/sneezing, viral upper respiratory infection, gastroenteritis, bacteremia, and diarrhea.
  • 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, an enzyme that reduces levels of neurotoxic sulfites.
  • Manufacturer: Alcami Carolinas Corporation



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