Nulibry

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

• Start NULIBRY if known or presumed MoCD Type A. Promptly discontinue if MoCD Type A is not confirmed by genetic testing. (
  2.1 Patient Selection

  Start NULIBRY if the patient has a diagnosis or presumptive diagnosis of MoCD Type A.

  In patients with a presumptive diagnosis of MoCD Type A, confirm the diagnosis of MoCD Type A immediately after initiation of NULIBRY treatment. In such patients, discontinue NULIBRY if the MoCD Type A diagnosis is not confirmed by genetic testing.
  )
• Reconstitute before use and complete infusion within 4 hours of reconstitution. (
  2.2 Important Administration Information

  • NULIBRY is intended for administration by a healthcare provider. If deemed appropriate by a healthcare provider, NULIBRY may be administered at home by the patient's caregiver. If NULIBRY can be administered by a caregiver/patient, advise them to read the detailed instructions on the preparation, administration, storage, and disposal of NULIBRY for caregivers
    [see Instructions for Use].
  • NULIBRY is for intravenous infusion only. Administer with non-DEHP tubing with a 0.2 micron filter. Do not mix NULIBRY with other drugs (note NULIBRY is reconstituted with Sterile Water for Injection, USP). Do not administer as an infusion with other drugs.
  • NULIBRY is given through an infusion pump at a rate of 1.5 mL per minute.
  • Dose volumes below 2 mL may require syringe administration through slow intravenous push.
  • Administration of NULIBRY must be completed within 4 hours of reconstitution
    [see Dosage and Administration ]
    .
  ,
  2.4 Preparation and Administration Instructions

  NULIBRY must be reconstituted prior to use. Use aseptic technique during preparation and follow these instructions:

  • Determine the total dose, number of vials needed, and total reconstituted dose volume based on the patient's weight and prescribed dose.
  • Remove the required number of vials from the freezer to allow them to reach room temperature (by hand warming for 3 to 5 minutes or exposing to ambient air for approximately 30 minutes).
  • Reconstitute each required NULIBRY vial with 5 mL of Sterile Water for Injection, USP. Gently swirl the vial continuously until the powder is completely dissolved. DO NOT shake. After reconstitution, the final concentration of NULIBRY reconstituted solution is 9.5 mg/5 mL (1.9 mg/mL).
  • Parenteral drug products should be inspected visually for particulate matter and discoloration prior to administration, whenever solution and container permit. Reconstituted NULIBRY is a clear and colorless to pale yellow solution. Do not use if there are particles present or if the solution is discolored.
  • Administer the total reconstituted dose.
  )
• Administer as an intravenous infusion once daily at a rate of 1.5 mL/minute with non-DEHP tubing with a 0.2 micron filter. Volumes below 2 mL may require syringe administration through slow intravenous push. (
  2.2 Important Administration Information

  • NULIBRY is intended for administration by a healthcare provider. If deemed appropriate by a healthcare provider, NULIBRY may be administered at home by the patient's caregiver. If NULIBRY can be administered by a caregiver/patient, advise them to read the detailed instructions on the preparation, administration, storage, and disposal of NULIBRY for caregivers
    [see Instructions for Use].
  • NULIBRY is for intravenous infusion only. Administer with non-DEHP tubing with a 0.2 micron filter. Do not mix NULIBRY with other drugs (note NULIBRY is reconstituted with Sterile Water for Injection, USP). Do not administer as an infusion with other drugs.
  • NULIBRY is given through an infusion pump at a rate of 1.5 mL per minute.
  • Dose volumes below 2 mL may require syringe administration through slow intravenous push.
  • Administration of NULIBRY must be completed within 4 hours of reconstitution
    [see Dosage and Administration ]
    .
  )
• See Full Prescribing Information for additional important preparation instructions and administration instructions. (
  2.2 Important Administration Information

  • NULIBRY is intended for administration by a healthcare provider. If deemed appropriate by a healthcare provider, NULIBRY may be administered at home by the patient's caregiver. If NULIBRY can be administered by a caregiver/patient, advise them to read the detailed instructions on the preparation, administration, storage, and disposal of NULIBRY for caregivers
    [see Instructions for Use].
  • NULIBRY is for intravenous infusion only. Administer with non-DEHP tubing with a 0.2 micron filter. Do not mix NULIBRY with other drugs (note NULIBRY is reconstituted with Sterile Water for Injection, USP). Do not administer as an infusion with other drugs.
  • NULIBRY is given through an infusion pump at a rate of 1.5 mL per minute.
  • Dose volumes below 2 mL may require syringe administration through slow intravenous push.
  • Administration of NULIBRY must be completed within 4 hours of reconstitution
    [see Dosage and Administration ]
    .
  )
• See the table below for the recommended dosage in patients less than one year of age. (
  2.3 Recommended Dosage and Administration

  Recommended Dosage and Administration in Patients Less Than One Year of Age (by gestational age)

  The recommended dosage regimen of NULIBRY in patients less than one year of age (by gestational age) is based on actual body weight as shown in Table 1.

  Table 1 Recommended Initial Dosage and Titration Schedule of NULIBRY for Patients Less Than One Year of Age by Gestational Age

  Titration Schedule	Preterm Neonates (Gestational Age Less than 37 Weeks)	Term Neonates (Gestational Age 37 Weeks and Above)
  Initial Dosage	0.4 mg/kg once daily	0.55 mg/kg once daily
  Dosage at Month 1	0.7 mg/kg once daily	0.75 mg/kg once daily
  Dosage at Month 3	0.9 mg/kg once daily	0.9 mg/kg once daily

  Recommended Dosage and Administration in Patients One Year of Age or Older

  For patients one year of age or older, the recommended dosage of NULIBRY is 0.9 mg/kg (based on actual body weight) administered as an intravenous infusion once daily.

  Recommendations for a Missed Dose

  If a NULIBRY dose is missed, administer the missed dose as soon as possible. Administer the next scheduled dose at least 6 hours after the administration of the missed dose.
  )

For injection: 9.5 mg of fosdenopterin, as a white to pale yellow lyophilized powder or cake in a single-dose vial for reconstitution.

There are no available data on NULIBRY use in pregnant women to evaluate for a drug-associated risk of major birth defects, miscarriage, or adverse maternal or fetal outcomes. Animal reproduction toxicology studies have not been conducted with fosdenopterin.

The estimated background risk of major birth defects and miscarriage for the indicated population is unknown. All pregnancies have a background risk of birth defect, loss, or other adverse outcomes. In the U.S. general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies are 2% to 4% and 15% to 20%, respectively.