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NULIBRY—shown to improve survival in patients with MoCD Type A

Finally, children who are diagnosed with molybdenum cofactor deficiency (MoCD) Type A have a fighting chance.1

NULIBRY is a cyclic pyranopterin monophosphate (cPMP) for patients with MoCD Type A. NULIBRY replaces the critical component your child needs to make molybdenum cofactor (MoCo) since children with MoCD Type A can't make it on their own.1

NULIBRY comes as a lyophilized powder or cake in vials that must stay frozen in a medical-grade freezer until ready to be used.1

NULIBRY must be prepared every day before you give it to your child. Before it is administered, it needs to be mixed or dissolved. This can only be done with Sterile Water for Injection and must be used within 4 hours of mixing. NULIBRY that has been mixed (dissolved) is given to your child through a port that is surgically placed under your child's skin at the hospital.1

Your child's healthcare provider will go over preparing and administering your child NULIBRY in detail with you before you leave the hospital. You can also download Full Prescribing Information, including Instructions for Use, for NULIBRY.

Children with MoCD Type A saw benefits from NULIBRY therapy1*

  • Improved overall survival as compared to patients not treated in the past1
  • Reduced levels of SSC, one of the compounds that builds up and is thought to cause seizures and severe brain abnormalities in patients with MoCD Type A1

*NULIBRY therapy was studied in 9 children who were confirmed to have MoCD Type A. The median time children remained on treatment was 4.3 years and ranged from 8 days to 5.6 years.1

Common side effects (in over 25% of children) included1†:

  • Catheter-related complications (89%)
  • Fever (78%)
  • Viral infections (56%)
  • Pneumonia (44%)
  • Ear infections (44%)
  • Vomiting (44%)
  • Coughing/sneezing (44%)
  • Common cold/flu-like infections (33%)
  • Stomach flu-like symptoms (33%)
  • Diarrhea (33%)
  • Bacteria in the blood (33%)

 Your child may have side effects from NULIBRY that are not on this list. Let your healthcare provider know about any side effects your child may be experiencing.

†Assessment of side effects for NULIBRY is based on data from open-label studies, Study 1 (8 patients) and Study 2 (1 patient), in children with a confirmed diagnosis of MoCD Type A in which children received NULIBRY.1

Patients treated with NULIBRY should avoid exposure to direct sunlight and artificial UV light exposure (ie, phototherapy) and adopt precautionary measures (eg, wear protective clothing and hats, use broad-spectrum sunscreen with high sun protection factor [SPF], and wear sunglasses when exposed to the sun). If photosensitivity occurs, caregivers/patients should seek medical attention immediately and consider a dermatological evaluation.1

The team at Sentynl Cares | NULIBRY is ready to assist you and your child throughout your journey. Contact the hotline with any questions or concerns at 1-888-251-2800, Monday-Friday, 8 am-8 pm ET.

Reference: 

  1. NULIBRY [prescribing information] https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=736aeea3-f206-454d-95d0-fd30964e8aab.
Preparation & Administration >

INDICATION AND IMPORTANT SAFETY INFORMATION

INDICATION

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

IMPORTANT SAFETY INFORMATION

WARNINGS AND PRECAUTIONS

Potential for Photosensitivity

NULIBRY can make the patient oversensitive to sunlight. NULIBRY-treated patients or their caregivers are advised to avoid or minimize patient exposure to sunlight and artificial UV light and adopt precautionary measures when exposed to the sun, including wearing protective clothing and sunglasses, and use broad-spectrum sunscreen with high SPF in patients 6 months of age and older. If photosensitivity occurs, caregivers/patients are advised to seek medical attention immediately and consider a dermatological evaluation.

SIDE EFFECTS

The most common side effects in NULIBRY-treated patients were infusion catheter–related complications, pyrexia (fever), viral infection, pneumonia, otitis media (ear infection), vomiting, cough/sneezing, viral upper respiratory infection (common cold/flu-like infection), gastroenteritis (stomach flu-like symptoms), diarrhea, and bacteremia (bacteria in the blood). Side effects for rcPMP-treated patients were similar to the NULIBRY-treated patients.

PATIENT COUNSELING INFORMATION

Please read the FDA-approved NULIBRY Prescribing Information and Instructions for Use and follow the instructions on how to prepare and administer NULIBRY.

NULIBRY has a potential for photosensitivity; see Warnings and Precautions. Seek medical attention immediately if the patient develops a rash or if they notice symptoms of photosensitivity reactions (redness, burning sensation of the skin, blisters).

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088.