Nulibry
Nulibry (fosdenopterin) is the first and only approved medication for the treatment of molybdenum cofactor deficiency (MoCD) Type A, a very rare genetic condition. It's typically given as an infusion through the veins (IV) once a day by a healthcare provider, but with proper training, some caregivers can give this medication to their child at home. Nulibry (fosdenopterin) can cause a lot of side effects, include infections, stomach problems, and redness and swelling at the injection site.
What is Nulibry (fosdenopterin)?
What is Nulibry (fosdenopterin) used for?
- To lower risk for death in people with molybdenum cofactor deficiency (MoCD) Type A
How Nulibry (fosdenopterin) works
If your child has MoCD Type A, there's a change in one of their genes that results in their body not being able to make certain compounds, such as cyclic pyranopterin monophosphate (cPMP), to help them get rid of certain toxic chemicals. As a result, the toxins build up in their body, particularly the brain.
Nulibry (fosdenopterin) is a cPMP analog, which means it acts just like cPMP. It treats MoCD Type A by replacing the missing compound to help your child lower the level of toxins in their body.
Drug facts
Side effects of Nulibry (fosdenopterin)
The following side effects may get better over time as your body gets used to the medication. Let your healthcare provider know immediately if you continue to experience these symptoms or if they worsen over time.
Common Side Effects
- Redness, swelling, infection, and other catheter-related complications (89%)
- Fever (78%)
- Viral infection (56%)
- Pneumonia (44%)
- Ear infection (44%)
- Vomiting (44%)
- Cough and sneezing (44%)
- Upper respiratory infection (33%)
- Stomach infection (33%)
- Diarrhea (33%)
- Bloodstream infection (33%)
Less Common Side Effects
- Stomach pain
- Flu infection
- Lower respiratory tract infection
- Viral infection in tonsils
- Pain in mouth and throat
- Rash
- Low red blood cell count (anemia)
- Eye swelling
- Seizure
- Agitation
The following Nulibry (fosdenopterin) side effects have also been reported
Side effects that you should report to your care team as soon as possible:
- Allergic reactions—skin rash, itching, hives, swelling of the face, lips, tongue, or throat
- Skin reactions on sun-exposed areas
Side effects that usually do not require medical attention (report these to your care team if they continue or are bothersome):
Pros and cons of Nulibry (fosdenopterin)
Pros
- Nulibry (fosdenopterin) is the only medication approved for MoCD Type A
- Improves survival for people with MoCD Type A
- Can be given at home after being properly trained by a healthcare professional
- Taken just once per day
- None of the components of Nulibry (fosdenopterin) are made with natural rubber latex.
Cons
- Needs to be given as an IV infusion or injection
- Lots of potential side effects, including catheter site redness and swelling, infections, and diarrhea
- Only studied in a small number of children
- Brand name only, which can be expensive
- Must be stored in the freezer
Pharmacist tips for Nulibry (fosdenopterin)
- Read the instructions or watch this video on how to give your child Nulibry (fosdenopterin). Before you give your child this medication at home, make sure your provider teaches you how to give Nulibry (fosdenopterin) so that you avoid accidentally hurting yourself or wasting the medication. Contact your provider if you have any questions on how to use this medication.
- Store unmixed Nulibry (fosdenopterin) vials in the freezer and keep the vials in the original carton away from light. When it's time to mix the medication, take the vial out of the freezer and allow it to reach room temperature. This can be done by rolling the vial(s) gently between your hands for 3-5 minutes or sitting at room temperature for about 30 minutes. Don't try to warm up the medication any other way (e.g., microwave, under hot water) because you might damage it.
- If you're giving your child Nulibry (fosdenopterin) at home, make sure to first gather the supplies you need to give the medication. Unopened Nulibry (fosdenopterin) comes as a powder in a vial. You need to mix it with 5 mL of sterile water, which is provided for you, before injection. Depending on your child's dose, you might need to use more than one vial to give the full dose.
- After mixing Nulibry (fosdenopterin) powder with sterile water, always check to make sure the medication is clear and colorless to pale yellow before using it. If it's cloudy, discolored, or has particles (solid pieces), don't use it. Contact your provider or pharmacist to see if it's possible to get a replacement. Don't throw away the vial because your pharmacist might ask you to return it.
- Nulibry (fosdenopterin) vials are single-dose only. After mixing, you must give the medication within 4 hours. Throw away the remainder of the medication after each use, even if there's still medication left.
- After giving your child the medication, throw away the syringe and needle in a sharps container or something made of heavy-duty plastic, such as an empty milk carton or laundry detergent bottle, to lower the risk of needle-stick injuries. Don't throw them away in your trash can. When your sharps container is almost full, close it tightly with the cap. For more information on how to throw away your sharps container safely and guidance for your specific state, visit the FDA's website.
- Fill out the treatment log that comes with Nulibry (fosdenopterin) after each time you give a dose as well as any time there are dose changes by your provider. This will help make sure you stay organized and are using the correct dose. Bring the log to each follow-up visit with your provider.
- Remember Nulibry (fosdenopterin) dose is based on your child's weight. Please measure your child's weight regularly and let your provider know of any changes so that if needed, your child's dose can be adjusted.
Common questions about Nulibry (fosdenopterin)
MoCD Type A is a rare genetic problem that's typically appears shortly after birth. If your baby has this condition, they can't make a compound called cPMP. Without cPMP, their body can't make a protein called molybdenum cofactor (MoCo), which helps them get rid of certain toxic chemicals. As the toxins build up in their body, they can experience severe brain problems. Signs and symptoms include seizures, trouble feeding, fussiness, and problems with muscle tone. Nulibry (fosdenopterin) helps because it acts like cPMP, which means it gets converted to MoCo and lowers the levels of certain toxic chemicals.
Nulibry (fosdenopterin) is typically given as an injection through the veins (IV) by a healthcare provider. However, you can give Nulibry (fosdenopterin) to your child at home if their healthcare provider says it's okay for you to do so. The medication is typically given through an infusion pump at a rate of 1.5 mL per minute. However, if the dose volume is below 2 mL, it's given as a slow IV push into your child's veins. Never inject Nulibry (fosdenopterin) into a muscle or under the skin. Never mix Nulibry (fosdenopterin) with any other IV medications.
Since MoCD Type A is a genetic problem, your child will likely need to take Nulibry (fosdenopterin) for their entire life, as long as it's helping their condition and they're not having severe side effects.
If your child misses a dose, give the dose as soon as you remember. Make sure that the next scheduled dose is given at least 6 hours after giving the missed dose so the two doses aren't given too close together.
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Risks and warnings for Nulibry (fosdenopterin)
Nulibry (fosdenopterin) can cause some serious health issues. This risk may be even higher for certain groups. If this worries you, talk to your doctor or pharmacist about other options.
Sensitivity to light
Nulibry (fosdenopterin) might make your child more sensitive to light. Make sure to avoid or lessen the time your child spends in direct sunlight or UV light by dressing them in protective clothing and hats, using sunscreen with a high SPF (especially for children at least 6 months of age and older), and putting sunglasses on them. If your child experience skin reactions such as redness, swelling, and flaky or peeling skin, get medical help right away.
Nulibry (fosdenopterin) dosage
Typical dosage for Nulibry (fosdenopterin)
Nulibry (fosdenopterin) is given as an injection through the veins (IV). It's typically given through an infusion pump at a rate of 1.5 mL per minute. However, if the dose volume is below 2 mL, it's given as a slow IV push into your child's veins. The dose is calculated based on your child's actual body weight.
Children age less than 1 year: The typical dose is based on the gestational age of your baby:
- Baby born premature (age less than 37 weeks): The typical starting dose is 0.4 mg/kg IV once per day. At month 1, the dose is raised to 0.7 mg/kg once per day. At month 3, the dose is raised to 0.9 mg/kg once per day.
- Baby born full term (age 37 weeks and older): The typical starting dose is 0.55 mg/kg IV once per day. At month 1, the dose is raised to 0.75 mg/kg once per day. At month 3, the dose is raised to 0.9 mg/kg once per day.
Children age 1 year and older: The typical dose is 0.9 mg/kg IV once per day.
Nulibry (fosdenopterin) images
