Treating Urea Cycle Disorders

Your UCD care team

People with urea cycle disorders (UCDs) usually work with a healthcare team that has several members. You may see a primary care doctor as well as a team of specialists at a metabolic center. These specialists may include a metabolic geneticist, a dietitian, nurses, a genetic counselor, a nurse practitioner, and a social worker.

Metabolic geneticists are specialists who treat disorders like UCDs. The metabolic geneticist, or one of his or her team members, will do ammonia level tests, create a UCD management plan, and make any changes to treatment. Your metabolic geneticist will help coordinate with other specialists, and can be the main point of contact for your primary care doctor. Your care team will also help with things like understanding genetics, UCDs, and family planning.

Managing your UCD

The dietary goal of managing a UCD is to control the amount of ammonia that is produced as the body breaks down protein. One way to produce less ammonia is to eat less protein. When living with a UCD, it's important to understand your protein limitations, especially since the right amount of protein is different for everyone. Limiting it too much may cause your body harm, too, because we need protein to grow and for other bodily functions. A dietitian will help you manage your low-protein diet. He or she will help you make a plan that works for your personal needs and restrictions.

Before making changes to your diet, you should speak with a dietitian to decide the right foods and portion sizes for you. A dietitian can help plan low-protein meals that meet your or your child's needs, and change them as your child grows. For recipes to help balance your daily protein needs and make it easier to prepare meals, sign up for our free low-protein cookbook.

Your care team will work to develop a customized treatment plan that's right for you or your child. This can include:

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  • A low-protein diet: Protein in the diet may be lowered by limiting or not eating protein-rich foods like meat, nuts, eggs, and cheese. There are also some fruits, vegetables, and grains that may have too much protein for some UCD patients. You should speak with your dietitian or medical team to develop a diet that is best for you. Special low-protein foods in moderation can also help keep protein within your limits. These can include low-protein pastas and breads, and special formulas. Your dietitian can teach you how to weigh or measure foods to make sure you eat the right amount of protein.

Citrulline and Arginine Supplements Icon Citrulline and Arginine Supplements Icon Citrulline and Arginine Supplements Icon
  • Amino acid supplements: Some nutrients, like certain amino acids, are found only in protein. Your dietitian may suggest daily dietary supplements to make sure you are getting all of the necessary vitamins and nutrients that may be missing from a diet limited in protein.

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  • Medicines: Sometimes a low-protein diet and supplements aren’t enough to keep ammonia levels low. In these cases, medicines called nitrogen scavengers (also referred to as “ammonia scavengers”) help remove ammonia from your blood, and may be added to your UCD treatment plan. RAVICTI® (glycerol phenylbutyrate) Oral Liquid is one nitrogen scavenger treatment option. Learn more about RAVICTI. You should not take RAVICTI if you are allergic to phenylbutyrate.

Even if the level of ammonia in your blood doesn’t get to a dangerously high level, any elevation of ammonia can still be harmful. That’s why one of the most important goals of managing a UCD is to keep the amount of ammonia in your blood at a safe and normal level. The most important thing you can do is stick to your UCD management plan to help keep your ammonia levels in check.

Managing Ammonia Levels Caused by UCDs

Learn about long-term ammonia control with RAVICTI

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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) Phenylacetate (PAA), a breakdown product of RAVICTI, may cause nervous system side effects.

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)
 Phenylacetate (PAA), a breakdown product of RAVICTI, 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

Your doctor may do blood tests to measure the amount of PAA in your blood during your treatment with RAVICTI.

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 for the acute treatment of hyperammonemia in people with UCD.
  • It is not known if RAVICTI is safe and effective for the treatment of N-acetylglutamate synthase (NAGS) deficiency.

Do not take RAVICTI if you are allergic to phenylbutyrate. Call your doctor or go to the nearest hospital emergency room if you have wheezing, shortness of breath, cough, low blood pressure, flushing, nausea or a rash while taking RAVICTI.

Before taking RAVICTI, tell your doctor about all of your medical conditions, including 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. If you become pregnant during treatment with RAVICTI, call Horizon at 1‐866‐479‐6742 to report the pregnancy.
  • 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
  • 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 may report side effects to FDA at 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) Phenylacetate (PAA), a breakdown product of RAVICTI, may cause nervous system side effects.

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)
 Phenylacetate (PAA), a breakdown product of RAVICTI, 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

Your doctor may do blood tests to measure the amount of PAA in your blood during your treatment with RAVICTI.

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 for the acute treatment of hyperammonemia in people with UCD.
  • It is not known if RAVICTI is safe and effective for the treatment of N-acetylglutamate synthase (NAGS) deficiency.

Do not take RAVICTI if you are allergic to phenylbutyrate. Call your doctor or go to the nearest hospital emergency room if you have wheezing, shortness of breath, cough, low blood pressure, flushing, nausea or a rash while taking RAVICTI.

Before taking RAVICTI, tell your doctor about all of your medical conditions, including 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. If you become pregnant during treatment with RAVICTI, call Horizon at 1‐866‐479‐6742 to report the pregnancy.
  • 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
  • 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 may report side effects to FDA at 1-800-FDA-1088.

For additional Important Safety Information, click here for the Medication Guide and discuss with your doctor.