UCD MENTORS: Support while taking RAVICTI® (glycerol phenylbutyrate) oral liquid

Talk to someone who’s been there

When you or a loved one is diagnosed with a urea cycle disorder (UCD), it can feel like your whole world has been turned upside down. You may have questions like: What now? How is this going to affect my life? Where do I go for advice? Who can I talk to? You'll be glad to know there is a community of people with UCDs who have been in your shoes and are happy to talk to you.

Meet the UCD mentors

When you or a loved one is diagnosed with a UCD, it can feel like your whole world has been turned upside down. You may have questions like: What now? How is this going to ae ct my life? Where do I go for advice? Who can I talk to? You’ll be glad to know there is a community of people with UCDs who have been in your shoes and are happy to talk to you.

Meet Amy

Meet Amy
Read transcript

Amy:

They always say it takes a village to raise a child. Raising a child with a rare disease, I always tell people in my case it takes a small army.

My name’s Amy, and I’m a UCD mentor.

I used to have hobbies. Now I have kids. We have 2 kids. Ah, TJ. And then there’s Mya. They are twins.

We first learned that Mya had a UCD when she was 6 months old. They always tell twin moms don’t compare your kids. But I had one, my son, who ate all the time and was healthy and happy, and I had my daughter, who was fussy all the time, and she would spit up, and I just knew something was wrong.

So I pushed the pediatrician and he did some tests. They told us, “It appears your daughter is in liver failure.” Then they determined it must have been a virus. They sent us home. In about a week, the doctors called me and said, “It’s possible it’s this really rare genetic disorder, this urea cycle disorder, so we’re gonna test her for that.” And we got the results about 3 days later.

So Mya has OTC deficiency. 

And I was like, okay, at least now we know what she has. And now we can deal with it.

The doctors and nurses are amazing, but you know your child best. You can’t be afraid to tell ‘em that. 

To be a mother of a child with a rare disease, you have to find your own voice. And you have to not be afraid to use it.

I always tell folks, you can’t give up. You absolutely cannot give up.

It’s gonna be hard. It’s gonna be exhausting, but you can do it. Your child can do it. And you’re not alone. You have resources. Don’t be afraid to reach out. And when people offer you help, take it.

I am a mentor. I’m here for you. Let’s talk.

 

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.

Meet Patricia

Meet Patricia
Read transcript

Patricia:

The challenges of being both the caregiver and a patient who has a urea cycle disorder, that can be quite a lot.

My name is Patricia. I’m a mom, I’m a wife, and I’m a caregiver. I have UCD, and my son also has urea cycle disorder.

We have ornithine transcarbamylase deficiency.

I first learned that I had a urea cycle disorder when my son was diagnosed.

When you first get that diagnosis of a urea cycle disorder, it feels like your world has been turned upside down. I have to say, though, that as devastating as the diagnosis was, it was a little bit of a relief. I felt hope, because for the first time so many unanswered things throughout my life and my family’s life finally made sense.

As a parent with a child with urea cycle disorder, you tend to have a little bit more on your plate.

The one thing I would say to that caregiver is don’t lose hope. You can do this. There are many people like myself who are here, ready to help. You’re not alone.

It’s hard at the end to take a little time away for yourself. But if you can’t take care of yourself, how can you take care of the ones you love?

I became a mentor because I feel like it’s the least I can do to pay it forward. I would love to be able to help somebody walk this walk.

I’m a UCD mentor. I’m here for you. Let’s talk.

 

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.

Meet David

Meet David
Read transcript

David:

We have hope. We have a community. The hardest thing about UCD is not knowing real people that are going through it. And, with the mentor program you’ll be able to talk to another person.

I’m David, and I’m a caregiver for my wife and son, who have urea cycle disorder.

I learned about Isaac having UCD when he was 8 years old. He started getting lethargic and started not feeling well. He started getting worse and worse. And so we drove him to the emergency room. And 4 hours later my son’s in critical condition. So it was at that point that he was diagnosed.

So we started getting treatment for it. And the doctor turns to my wife and goes, “And you? Has anyone treated you?” 

Because it’s X-linked. It’s handed from moms down to the children.

And all of a sudden, it’s just like, oh, my gosh, this is what we’ve been wrestling with. It was, it was like the world opened up. By getting the diagnosis. It sounds weird, because they’re saying you’re sick. But to get the diagnosis was a relief.

There’s no one who really understands unless someone that has done that and been there. And that’s why I’m making myself available if some people just need to talk. If nothing else, I understand.

My son’s now in college. My wife is getting more lovely every year. And we’ve been at it for 30 years, we’re looking forward to what’s in front of us.

We’ll treat this every single day. But we won’t let it conquer us.

I am a mentor. I am here for you. Let’s talk.

 

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.

Meet Isaac

Meet Isaac
Read transcript

Isaac:

Growing up with a UCD was interesting to say the least. I got a much different perspective than a lot of other kids. I always had to be careful. I had to stay in. I was homeschooled. So, I’d say I got a lot more creativity than most people.

I’m Isaac, and I’m living with OTC.

My family consists of me, my father, David, and my mother, Patricia.

Right now I’m just taking the core program in a community college in order to take all the basic classes like math, history, English, and all that.

I’m hoping to get into the video game design business. So I’m hoping to become a game designer.

I learned the hard way that I had UCD. It’s just a whole series of events that led to me actually falling into a coma that lasted for a week straight, and when I woke up, I was told that I had UCD.

It’s something I’ve been living with my entire life. It’s part of my everyday routine. At some point you just say, “Well, just bring on life. I’m ready to take you on.”

I definitely think when I started to take care of myself, my parents felt a lot more confident in letting me do my own thing, that I was more of an adult. I was proving that I was able to take care of myself, handle myself, I knew what I was doing.

You’re your own biggest ally in this.

I became a UCD Mentor because I feel like there are people out there that do need help and need to have someone to rely on and to give them some advice and all that. So with the experience I had, I figured it might be helpful for me to throw my hat into the ring.

I’m a mentor. I’m here for you. Let’s talk.

 

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.

Find more RAVICTI resources and stories from other patients and caregivers

See more

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.