What Is a UCD?

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USE and IMPORTANT SAFETY INFORMATION

What is the most important 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.

Amy:

Mya was diagnosed at 6 months old. Our first step was low-protein diet. And she was just on that until about a year. At a year, we added some supplements. At 2 years, we had to add a medication, an ammonia scavenger.

The ammonia scavenger she was on could leave a bad taste in the mouth.

It was challenging to just flat out get her to take it.

And I thought there has to be another treatment. I was willing to try something new.

So I went to the Internet, and I searched and I searched, and I found RAVICTI. And I thought, I have to get my child on this.

I did talk to my doctor about starting RAVICTI.

I had found most of the information online, so I pretty much sent him a link and said, "Here's this information." And he responded, "Great. I support you." RAVICTI may not be a fit for everybody. For Mya specifically, it is convenient because it’s tasteless, it’s odorless. Mya really doesn’t mind taking it.

For us, starting RAVICTI was pretty easy.

It was easy for her to take and the routine was the same, cause she still got the RAVICTI at the same time every day that she got the other one. It was just a different medication.

Every family’s experience is different, but for us Mya has been on RAVICTI for almost 6 years and we continue to like the convenience.

USE and IMPORTANT SAFETY INFORMATION

What is the most important 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:

• 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
• 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, see the Medication Guide available at RAVICTI.com and discuss with your doctor.