FDA approved Nulibry (cyclic pyranopterin monophosphate) for Molybdenum Cofactor Deficiency Type AFDA approved first drug for rare genetic disorder MoCD Type A.

Nulibry is cyclic pyranopterin monophosphate (cPMP), a precursor in the biosynthesis of molybdenum cofactor. In MoCD Type A, cPMP production is deficient, leading to accumulation of toxic metabolites. Nulibry replaces cPMP, restoring molybdenum cofactor synthesis and reducing mortality risk.

Nulibry is indicated to reduce the risk of mortality in patients with molybdenum cofactor deficiency (MoCD) Type A. Treatment should be initiated in patients with known or presumptive diagnosis of MoCD Type A. If diagnosis is presumptive, it must be confirmed by genetic testing immediately after initiation; Nulibry should be discontinued if MoCD Type A is not confirmed.

Nulibry is for intravenous infusion only. For patients less than one year of age, dosing is based on gestational age: preterm neonates (gestational age <37 weeks) start at 0.4 mg/kg once daily, titrating to 0.9 mg/kg by month 3; term neonates (≥37 weeks) start at 0.55 mg/kg once daily, titrating to 0.9 mg/kg by month 3. For patients one year or older, the recommended dose is 0.9 mg/kg once daily. Reconstitute each vial with 5 mL Sterile Water for Injection (final concentration 1.9 mg/mL). Administer at 1.5 mL/minute using non-DEHP tubing with a 0.2 micron filter. Complete infusion within 4 hours of reconstitution. For missed doses, administer as soon as possible and space next dose at least 6 hours apart.

The efficacy of Nulibry was established based on data from three clinical studies (Studies 1, 2, and 3) compared to data from a natural history study. Study 1 (NCT02047461) was a prospective, open-label, single-arm, dose escalation study. Detailed efficacy results are not available in the label.

Not reported in label.

Nulibry is the first FDA-approved therapy for MoCD Type A. It is indicated to reduce mortality risk and should be initiated promptly upon diagnosis or presumptive diagnosis, with genetic confirmation required. It is administered intravenously once daily with dose adjustments for age and gestational age.