RAVICTI for Newborns, Babies, and Toddlers

Learning that your baby has a urea cycle disorder (UCD) can be scary. A baby with a UCD needs long-term treatment to keep ammonia in the body at a safe level. This is why choosing the right medicine is so important. Take this survey to find out if RAVICTI® (glycerol phenylbutyrate) Oral Liquid may be right for your baby and watch this video to learn about using RAVICTI in babies 2 months of age and older.

Newborns (0-2 months of age)

RAVICTI® (glycerol phenylbutyrate) Oral Liquid is a ready‐to‐use liquid medicine that’s FDA‐approved to treat most types of UCDs in newborns whose UCD cannot be managed by diet and supplements alone. In a clinical study that lasted up to 24 months, RAVICTI helped keep ammonia levels in a normal range* for newborns from 0‐2 months of age.

  • 16 newborns less than 2 months of age took part in the clinical study
  • 5 of 16 newborns (31%) who took part in the clinical study reported a total of 7 hyperammonemic crises (extremely high levels of ammonia in the blood)
  • Mean normalized ammonia levels in newborn patients at months 1 through 6 and 6 through 12 were a range of 35 through 94 μmol/L during treatment with RAVICTI.

*Range of 28–57 μmol/L.

In an infant who is breastfeeding, give RAVICTI just prior to breastfeeding.

The most common side effects of RAVICTI in children less than 2 months of age include:  vomiting, rash, gastroesophageal reflux, increased levels of liver enzymes in the blood, decreased appetite and reduced food intake, low red blood cell count (anemia), cough, loss of too much body fluid (dehydration), too much acid in the blood (acidosis), high blood platelet count (thrombocytosis), low blood platelet count (thrombocytopenia), low blood neutrophil count (type of white blood cell) (neutropenia), high white blood cell count (lymphocytosis), diarrhea, gas, constipation, fever, drowsiness (lethargy), irritability, agitation.

Babies and toddlers (2 months to 2 years of age)

RAVICTI is the only ready-to-use liquid medicine that’s FDA-approved to treat most types of UCDs for babies and toddlers. You can watch this video to learn about using RAVICTI in babies age 2 months to 2 years.

In clinical studies that lasted up to 18 months, RAVICTI helped keep ammonia levels in a normal range* for young children ages 2 months to younger than 2 years.

  • 17 babies and toddlers took part in the clinical studies
  • 7 of the 17 babies and toddlers (41%) who took part in the clinical studies reported a total of 11 hyperammonemic crises
  • The mean normalized ammonia levels in babies and toddlers (2 months to 2 years of age) never exceeded 99 μmol/L during treatment with RAVICTI, with the majority of values well below this level

*Range of 24–48 μmol/L.

Young child with a UCD,  playing outside with bubbles
Read transcript

Finding out that your baby or toddler has a urea cycle disorder, also called a “UCD”, can be scary and overwhelming. There is a lot of information to process and understand.

One thing you’ll learn right away is the importance of managing the amount of ammonia in your child’s blood.

There are several things you can do to help prevent your child’s blood ammonia levels from getting too high, like feeding them a low-protein diet and monitoring their activity.

There are also treatments that can help keep your child’s ammonia levels under control over a long period of time.

RAVICTI (glycerol phenylbutyrate) Oral Liquid is a treatment option for babies and toddlers.

Studies have shown that RAVICTI helped manage ammonia levels in babies and toddlers younger than 2 years of age.

These studies included babies and toddlers who had been using other medicine to treat their UCDs, as well as toddlers who had not been using any medicine at all.

RAVICTI is a prescription medicine, and it is the only FDA-approved liquid medicine of its kind.

RAVICTI is taken by mouth, and can also be given through a feeding tube, if necessary. It is taken 3 or more times a day with food or formula and has little to no taste. In babies who are breastfeeding, RAVICTI should be given just before breastfeeding.

Babies and toddlers using RAVICTI must eat a low-protein diet and, in many cases, take dietary supplements to help manage ammonia levels.

RAVICTI should not be used in children of any age if they are allergic to phenylbutyrate.

Managing a UCD requires some lifestyle changes, but there are treatment options available to help to keep your child’s ammonia at a safe level over time.

If your baby or toddler has been diagnosed with a UCD, talk to your doctor to learn more about how RAVICTI might help you and your family.

 

IMPORTANT SAFETY INFORMATION

What is the most important safety information I should know about RAVICTI?

RAVICTI may cause serious side effects, including:
Nervous system side effects (Neurotoxicity). The breakdown of RAVICTI produces the byproduct phenylacetate (PAA), which may cause nervous system side effects. 
Call your doctor or get medical help right away if you have any of these symptoms while taking RAVICTI: 
  • sleepiness 
  • lightheadedness 
  • change in taste 
  • problems with hearing 
  • confusion 
  • problems with memory
  • worsening of numbness, tingling, or burning in your hands or feet  
  • headache 
  • feeling very tired (fatigue) 
  • nausea 
  • vomiting 

Who should not take RAVICTI?

Do not take RAVICTI if you are allergic to phenylbutyrate. Call your doctor or go to the nearest hospital emergency room if you experience any of the following symptoms of an allergic reaction while taking RAVICTI:

  • wheezing
  • shortness of breath
  • cough
  • low blood pressure
  • flushing
  • nausea
  • skin rash

 

What should I tell my doctor before taking RAVICTI?

Tell your doctor about any medical conditions and if you:

  • Have liver or kidney problems. 
  • Have pancreas or bowel (intestine) problems. 
  • Are pregnant or plan to become pregnant. It is not known if RAVICTI will harm your unborn baby. 
    • Pregnancy Registry: There is a Pregnancy Registry for women who take RAVICTI just before becoming pregnant or who become pregnant during treatment with RAVICTI. The purpose of this registry is to collect information about the health of you and your baby. Talk to your doctor about how you can join the Pregnancy Registry. For more information about this registry, call 1-855-823-2595 or visit www.ucdregistry.com.
  • Are breastfeeding or plan to breastfeed. It is not known if RAVICTI passes into your breast milk. Breastfeeding is not recommended during treatment with RAVICTI. Talk to your doctor about the best way to feed your baby if you take RAVICTI. 

What are possible side effects of RAVICTI?

RAVICTI may cause serious side effects, including:

  • See What is the most important information I should know about RAVICTI?

 The most common side effects of RAVICTI in adults include:

  • diarrhea
  • gas
  • headache
  • abdomen (stomach) pain
  • vomiting
  • tiredness
  • decreased appetite
  • indigestion or heartburn 

The most common side effects of RAVICTI in children 2 years to 17 years of age include:

  • upper abdomen (stomach) pain
  • rash
  • nausea
  • vomiting
  • diarrhea
  • decreased appetite
  • headache 

The most common side effects of RAVICTI in children 2 months to less than 2 years of age include:

  • low white blood cell count (neutropenia)
  • vomiting
  • diarrhea
  • fever
  • reduced food intake
  • cough
  • stuffy nose
  • runny nose
  • skin rash
  • small round bumps on the skin 

The most common side effects of RAVICTI in children less than 2 months of age include:

  • vomiting
  • rash
  • gastroesophageal reflux
  • increased levels of liver enzymes in the blood
  • decreased appetite and reduced food intake
  • low red blood cell count (anemia)
  • cough
  • loss of too much body fluid (dehydration)
  • too much acid in the blood (acidosis)
  • high blood platelet count (thrombocytosis)
  • low blood platelet count (thrombocytopenia)
  • low blood neutrophil count (type of white blood cell) (neutropenia)
  • high white blood cell count (lymphocytosis)
  • diarrhea
  • gas
  • constipation
  • fever
  • drowsiness (lethargy)
  • irritability
  • agitation

These are not all of the possible side effects of RAVICTI.  Call your doctor for medical advice about side effects.

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

For additional important safety information, see the Medication Guide available at RAVICTI.com and discuss with your doctor.

Watch this video to learn how RAVICTI can help control ammonia levels for babies and toddlers 2 months to 2 years of age.

The most common side effects of RAVICTI in children 2 months to less than 2 years of age include: low white blood cell count (neutropenia), vomiting, constipation, diarrhea, fever, reduced food intake, cough, stuffy nose, runny nose, skin rash, and small round bumps on the skin. Learn more about important safety information and side effects.

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"As the mother of a child with a rare
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 – Amy | Caregiver

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USE and IMPORTANT SAFETY INFORMATION

What is the most important safety information I should know about RAVICTI? RAVICTI may cause serious side effects, including: Nervous system side effects (Neurotoxicity). The breakdown of RAVICTI produces the byproduct phenylacetate (PAA), which may cause nervous system side effects.

What is RAVICTI?

RAVICTI (glycerol phenylbutyrate) Oral Liquid is a prescription medicine used for long-term management of high blood levels of ammonia (hyperammonemia) caused by a condition called a urea cycle disorder (UCD). RAVICTI should be used if the UCD cannot be managed with a low-protein diet and dietary supplements alone. RAVICTI must be used along with a low-protein diet and in some cases, dietary supplements.

RAVICTI is not used to treat extremely high levels of ammonia in the blood (hyperammonemic crisis) in people with UCDs.

It is not known if RAVICTI is safe and effective for the treatment of N-acetylglutamate synthase (NAGS) deficiency.

IMPORTANT SAFETY INFORMATION

What is the most important safety information I should know about RAVICTI?

RAVICTI may cause serious side effects, including:

Nervous system side effects (Neurotoxicity). The breakdown of RAVICTI produces the byproduct phenylacetate (PAA), which may cause nervous system side effects.

Call your doctor or get medical help right away if you have any of these symptoms while taking RAVICTI:

  • sleepiness
  • lightheadedness
  • change in taste
  • problems with hearing
  • confusion
  • problems with memory
  • worsening of numbness, tingling, or burning in your hands or feet
  • headache
  • feeling very tired (fatigue)
  • nausea
  • vomiting

Who should not take RAVICTI?

Do not take RAVICTI if you are allergic to phenylbutyrate. Call your doctor or go to the nearest hospital emergency room if you experience any of the following symptoms of an allergic reaction while taking RAVICTI:

  • wheezing
  • shortness of breath
  • cough
  • low blood pressure
  • flushing
  • nausea
  • skin rash

What should I tell my doctor before taking RAVICTI?

Tell your doctor about any medical conditions and if you:

  • Have liver or kidney problems.
    • Have pancreas or bowel (intestine) problems.
      • Are pregnant or plan to become pregnant. It is not known if RAVICTI will harm your unborn baby.
        • Pregnancy Registry: There is a Pregnancy Registry for women who take RAVICTI just before becoming pregnant or who become pregnant during treatment with RAVICTI. The purpose of this registry is to collect information about the health of you and your baby. Talk to your doctor about how you can join the Pregnancy Registry. For more information about this registry, call 1-855-823-2595 or visit www.ucdregistry.com.
      • Are breastfeeding or plan to breastfeed. It is not known if RAVICTI passes into your breast milk. Breastfeeding is not recommended during treatment with RAVICTI. Talk to your doctor about the best way to feed your baby if you take RAVICTI.

        What are possible side effects of RAVICTI?

        RAVICTI may cause serious side effects, including:

        • See "What is the most important information I should know about RAVICTI?"

        The most common side effects of RAVICTI in adults include:

        • diarrhea
        • gas
        • headache
        • abdomen (stomach) pain
        • vomiting
        • tiredness
        • decreased appetite
        • indigestion or heartburn

        The most common side effects of RAVICTI in children 2 years to 17 years of age include:

        • upper abdomen (stomach) pain
        • rash
        • nausea
        • vomiting
        • diarrhea
        • decreased appetite
        • headache

        The most common side effects of RAVICTI in children 2 months to less than 2 years of age include:

        • low white blood cell count (neutropenia)
        • vomiting
        • constipation
        • diarrhea
        • fever
        • reduced food intake
        • cough
        • runny nose
        • stuffy nose
        • skin rash
        • small round bumps on the skin

        The most common side effects of RAVICTI in children less than 2 months of age include:

        • vomiting
        • rash
        • gastroesophageal reflux
        • increased levels of liver enzymes in the blood
        • decreased appetite and reduced food intake
        • low red blood cell count (anemia)
        • cough
        • loss of too much body fluid (dehydration)
        • too much acid in the blood (acidosis)
        • high blood platelet count (thrombocytosis)
        • low blood platelet count (thrombocytopenia)
        • low blood neutrophil count (type of
          white blood
          cell) (neutropenia)
        • high white blood cell count (lymphocytosis)
        • diarrhea
        • gas
        • constipation
        • fever
        • drowsiness (lethargy)
        • irritability
        • agitation

        These are not all of the possible side effects of RAVICTI. Call your doctor for medical advice about side effects.

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

        For additional important safety information, click here for the Medication Guide and discuss with your doctor.

        USE and IMPORTANT SAFETY INFORMATION

        What is the most important safety information I should know about RAVICTI? RAVICTI may cause serious side effects, including: Nervous system side effects (Neurotoxicity). The breakdown of RAVICTI produces the byproduct phenylacetate (PAA), which may cause nervous system side effects.

        What is RAVICTI?

        RAVICTI (glycerol phenylbutyrate) Oral Liquid is a prescription medicine used for long-term management of high blood levels of ammonia (hyperammonemia) caused by a condition called a urea cycle disorder (UCD). RAVICTI should be used if the UCD cannot be managed with a low-protein diet and dietary supplements alone. RAVICTI must be used along with a low-protein diet and in some cases, dietary supplements.

        RAVICTI is not used to treat extremely high levels of ammonia in the blood (hyperammonemic crisis) in people with UCDs.

        It is not known if RAVICTI is safe and effective for the treatment of N-acetylglutamate synthase (NAGS) deficiency.

        IMPORTANT SAFETY INFORMATION

        What is the most important safety information I should know about RAVICTI?

        RAVICTI may cause serious side effects, including:

        Nervous system side effects (Neurotoxicity). The breakdown of RAVICTI produces the byproduct phenylacetate (PAA), which may cause nervous system side effects.

        Call your doctor or get medical help right away if you have any of these symptoms while taking RAVICTI:

        • sleepiness
        • lightheadedness
        • change in taste
        • problems with hearing
        • confusion
        • problems with memory
        • worsening of numbness, tingling, or burning in your hands or feet
        • headache
        • feeling very tired (fatigue)
        • nausea
        • vomiting

        Who should not take RAVICTI?

        Do not take RAVICTI if you are allergic to phenylbutyrate. Call your doctor or go to the nearest hospital emergency room if you experience any of the following symptoms of an allergic reaction while taking RAVICTI:

        • wheezing
        • shortness of breath
        • cough
        • low blood pressure
        • flushing
        • nausea
        • skin rash

        What should I tell my doctor before taking RAVICTI?

        Tell your doctor about any medical conditions and if you:

        • Have liver or kidney problems.
          • Have pancreas or bowel (intestine) problems.
            • Are pregnant or plan to become pregnant. It is not known if RAVICTI will harm your unborn baby.
              • Pregnancy Registry: There is a Pregnancy Registry for women who take RAVICTI just before becoming pregnant or who become pregnant during treatment with RAVICTI. The purpose of this registry is to collect information about the health of you and your baby. Talk to your doctor about how you can join the Pregnancy Registry. For more information about this registry, call 1-855-823-2595 or visit www.ucdregistry.com.
            • Are breastfeeding or plan to breastfeed. It is not known if RAVICTI passes into your breast milk. Breastfeeding is not recommended during treatment with RAVICTI. Talk to your doctor about the best way to feed your baby if you take RAVICTI.

              What are possible side effects of RAVICTI?

              RAVICTI may cause serious side effects, including:

              • See "What is the most important information I should know about RAVICTI?"

              The most common side effects of RAVICTI in adults include:

              • diarrhea
              • gas
              • headache
              • abdomen (stomach) pain
              • vomiting
              • tiredness
              • decreased appetite
              • indigestion or heartburn

              The most common side effects of RAVICTI in children 2 years to 17 years of age include:

              • upper abdomen (stomach) pain
              • rash
              • nausea
              • vomiting
              • diarrhea
              • decreased appetite
              • headache

              The most common side effects of RAVICTI in children 2 months to less than 2 years of age include:

              • low white blood cell count (neutropenia)
              • vomiting
              • constipation
              • diarrhea
              • fever
              • reduced food intake
              • cough
              • runny nose
              • stuffy nose
              • skin rash
              • small round bumps on the skin

              The most common side effects of RAVICTI in children less than 2 months of age include:

              • vomiting
              • rash
              • gastroesophageal reflux
              • increased levels of liver enzymes in the blood
              • decreased appetite and reduced food intake
              • low red blood cell count (anemia)
              • cough
              • loss of too much body fluid (dehydration)
              • too much acid in the blood (acidosis)
              • high blood platelet count (thrombocytosis)
              • low blood platelet count (thrombocytopenia)
              • low blood neutrophil count (type of
                white blood
                cell) (neutropenia)
              • high white blood cell count (lymphocytosis)
              • diarrhea
              • gas
              • constipation
              • fever
              • drowsiness (lethargy)
              • irritability
              • agitation

              These are not all of the possible side effects of RAVICTI. Call your doctor for medical advice about side effects.

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

              For additional important safety information, click here for the Medication Guide and discuss with your doctor.