Medical Editor: John P. Cunha, DO, FACOEP
What Is Nulibry?
Nulibry (fosdenopterin) is cyclic pyranopterin monophosphate (cPMP) indicated to reduce the risk of mortality in patients with molybdenum cofactor deficiency (MoCD) Type A.
What Are Side Effects of Nulibry?
Side effects of Nulibry include:
- Nulibry (fosdenopterin) for Injectioncatheter-related complications,
- viral infection,
- middle ear infection,
- viral upper respiratory infection,
- bacteremia, and
Seek medical care or call 911 at once if you have the following serious side effects:
- Serious eye symptoms such as sudden vision loss, blurred vision, tunnel vision, eye pain or swelling, or seeing halos around lights;
- Serious heart symptoms such as fast, irregular, or pounding heartbeats; fluttering in your chest; shortness of breath; and sudden dizziness, lightheadedness, or passing out;
- Severe headache, confusion, slurred speech, arm or leg weakness, trouble walking, loss of coordination, feeling unsteady, very stiff muscles, high fever, profuse sweating, or tremors.
This document does not contain all possible side effects and others may occur. Check with your physician for additional information about side effects.
Dosage for Nulibry
The dose of Nulibry is administered as an intravenous infusion once daily at a rate of 1.5 mL/minute with non-DEHP tubing with a 0.2-micron filter.
Nulibry In Children
Safety and effectiveness of Nulibry for the treatment of MoCD Type A have been established in pediatric patients starting from birth.
What Drugs, Substances, or Supplements Interact with Nulibry?
Nulibry may interact with other medicines.
Tell your doctor all medications and supplements you use.
Nulibry During Pregnancy and Breastfeeding
Tell your doctor if you are pregnant or plan to become pregnant before using Nulibry; it is unknown how it would affect a fetus. It is unknown if Nulibry passes into breast milk. Consult your doctor before breastfeeding.
Our Nulibry (fosdenopterin) for Injection, for Intravenous Use Side Effects Drug Center provides a comprehensive view of available drug information on the potential side effects when taking this medication.
This is not a complete list of side effects and others may occur. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.
Clinical Trials Experience
Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice.
Overview Of Safety Evaluation
The safety of NULIBRY was assessed in 37 pediatric patients and healthy adults who received at least one intravenous infusion of NULIBRY or an E. coli derived non-salt, anhydrous form of cPMP (recombinant cPMP or rcPMP, which has the same active moiety and therefore the same biologic activity as NULIBRY). Of these 37 patients/healthy adults, 13 were pediatric patients with MoCD Type A in Studies 1, 2, and 3 [see Clinical Studies], 6 were pediatric patients with presumptive MoCD Type A but who were later confirmed to not have MoCD Type A, and 18 were healthy adults (without MoCD Type A) in a Phase 1 study.
Assessment of adverse reactions for NULIBRY is based on data from two open-label, single-arm studies, Study 1 (n=8) and Study 2 (n=1), in patients with a confirmed diagnosis of MoCD Type A (8 of the 9 patients were previously treated with rcPMP). In these studies, patients received a daily intravenous infusion of NULIBRY. The median exposure to NULIBRY was 4.3 years and ranged from 8 days to 5.6 years [see Clinical Studies]. In these studies, 44% of patients were males and 56% were females, 67% were White and 33% were Asian. The mean age was 14 days and ranged from 1 day to 69 days at time of first infusion.
Table 2 presents the most common adverse reactions that occurred in NULIBRY-treated patients in Studies 1 and 2.
Table 2 Common Adverse Reactions Reported in Two or More NULIBRY-Treated Patients with MoCD Type A (Studies 1 and 2)
|Adverse Reactions||NULIBRY-Treated Patients (N=9)
|Catheter-related complications1||8 (89%)|
|Viral infection||5 (56%)|
|Otitis Media||4 (44%)|
|Upper viral respiratory infection||3 (33%)|
|Abdominal pain||2 (22%)|
|Lower respiratory tract infection||2 (22%)|
|Viral tonsillitis||2 (22%)|
|Oropharyngeal pain||2 (22%)|
|Rash maculo-papular||2 (22%)|
|Eye swelling||2 (22%)|
|Abbreviations: MoCD = molybdenum cofactor deficiency
1 Catheter-related complications included complication associated with device, catheter site abscess, catheter site discharge, catheter site extravasation, catheter site pain, catheter site infection, catheter site inflammation, device dislocation, device leakage, device occlusion, and vascular device infection.
Safety data are also available from 10 patients with MoCD Type A who received rcPMP in Study 3 (an observational study) [see Clinical Studies]. The median time on rcPMP treatment was 1.5 years and ranged from 6 days to 4.4 years. In Study 3, the patient population was evenly distributed between males and females with a mean age of 18 days (range 1, 69) at time of first infusion, 70% were White, and 30% were Asian.
In Study 3, one patient died of necrotizing enterocolitis. Adverse reactions for the rcPMP-treated patients were similar to the NULIBRY-treated patients, except for the following additional adverse reactions that were reported in more than one patient: sepsis, oral candidiasis, varicella, fungal skin infection, and eczema.
No Information Provided
Read the entire FDA prescribing information for Nulibry (Fosdenopterin for Injection)
© Nulibry Patient Information is supplied by Cerner Multum, Inc. and Nulibry Consumer information is supplied by First Databank, Inc., used under license and subject to their respective copyrights.
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