Learn about RAVICTI® (glycerol phenylbutyrate) Oral Liquid

If you or a loved one is living with a urea cycle disorder (UCD), RAVICTI may be able to help. Click one of the topics below or scroll down to learn more about

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What is RAVICTI?

RAVICTI is a treatment for UCDs

When you have a UCD, your body doesn’t get rid of a waste product called ammoniaA waste product of protein digestion that is removed by the urea cycle process. the way it should. When too much ammonia builds up in your body, it can be dangerous.

The good news is that RAVICTI can be used in adults and children 2 years of age and older for the long-term management of high blood levels of ammonia caused by a condition called a UCD. It is the only FDA-approved liquid medicine for UCDs that:

  • Can be taken by mouth
  • Has little-to-no taste
  • Along with a low-protein diet, can keep your ammonia at safe levels

Use RAVICTI only if you cannot manage your UCD with a low-protein diet and dietary supplementsEssential amino acids like arginine or citrulline may be added depending on the urea cycle defect. alone. RAVICTI should be used along with a low-protein diet and, in some cases, dietary supplements.

RAVICTI can be used in people with different UCDs, excluding N-acetylglutamate synthase (NAGS) deficiency

Although you or your child have been diagnosed with a UCD, the name that is used to refer to your UCD is based on the type of deficiencyA lower amount than necessary for functioning. you have.

UCD DEFICIENCIES TREATED BY RAVICTI
Ornithine transcarbamylase (OTC)
Argininosuccinate synthetase (ASS) or citrullinemia I
Argininosuccinate lyase (ASL) or argininosuccinic aciduria
Carbamyl phosphate synthetase (CPS1)
Arginase (ARG) or hyperargininemia
Ornithine translocase (ORNT1); also called hyperornithinemia-hyperammonemia-homocitrullinuria (HHH) syndrome
Citrin (aspartate glutamate translocase)

It is not known if RAVICTI is safe and effective in children who are less than 2 years of age or in people who have an N-acetylglutamate synthase (NAGS) deficiency.

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What are the benefits of RAVICTI?

The importance of ammonia control

One goal of managing a UCD is to lower the amount of ammoniaA waste product of protein digestion that is removed by the urea cycle process. in your blood to a safe and normal level. If ammonia levels are too high, they can lead to dangerous effects on your body and brain.

Ammonia levels can go up and down throughout the day. Even if your ammonia levels go up just a little bit, over time, serious health problems may occur. As part of the long-term management of your UCD, it is important to make sure your ammonia is at a safe and normal level. This is why your doctor or nurse will check your blood ammonia levels frequently.

How RAVICTI can help

In clinical studies, people who received RAVICTI for the treatment of their UCDs had the following results:

On RAVICTI, average blood ammonia levels in people 2 years of age and older were controlled at safe and normal levels for 24 hours.

 

 

On RAVICTI, average blood ammonia levels stayed within a normal range during the treatment year.

  • In adults, average blood ammonia levels were within the range of 6 to 30 µmol/L. This range was considered normal during the treatment year.
    • 7 out of 51 adults (14%) reported a total of 10 hyperammonemic crises.
  • In children 6 to 17 years of age, average blood ammonia levels were within the range of 17 to 23 µmol/L. This range was considered normal during the treatment year.
    • 5 out of 26 children 6 to 17 years of age (19%) reported a total of 5 hyperammonemic crises.

After using RAVICTI for 2 weeks, caregivers and patients were asked if they wanted to continue using RAVICTI for up to a year:

    • 91% of adults and their caregivers chose to continue with RAVICTI.
    • 100% of children 6 to 17 years of age and their caregivers chose to continue with RAVICTI.

Based on your ammonia level, you could experience varying symptoms and consequences.
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How Does RAVICTI work?

RAVICTI is the only ammonia-removing medicine available as an oral liquid

RAVICTI is a liquid prescription medicine that:

  • Can be taken by mouth
  • Has little-to-no taste
  • Does not have pills or powder that require any preparation

RAVICTI is more evenly released throughout the day

What makes RAVICTI unique from other ammoniaA waste product of protein digestion that is removed by the urea cycle process.-removing medicines is that the active ingredient is released slowly in your body, mostly in the small intestine. This means RAVICTI stays active in your body for a longer period of time while getting rid of unwanted ammonia.

RAVICTI has been studied over a long period of time

RAVICTI is a UCD treatment that has been studied by doctors and researchers for up to a year in adults and children 6 years of age and older. The FDA reviewed the results of these studies and approved RAVICTI as a safe and effective treatment for UCDs.

How do I take RAVICTI?

RAVICTI is easy to take

RAVICTI is a liquid prescription medicine that:

  • Can be taken by mouth
  • Has little-to-no taste
  • Does not have pills or powder that require preparation

Take RAVICTI with breakfast, lunch, and dinner

RAVICTI is usually taken 3 times a day with meals. You should take or give RAVICTI exactly as your doctor has prescribed it. Do not change your dose of RAVICTI without a doctor’s permission. Your doctor may change your dose if needed. If you take too much RAVICTI, call your doctor and go to the nearest hospital emergency room right away.

The maximum approved daily dose of RAVICTI is 17.5 mL for both adults and children.

RAVICTI comes with dosing tools for easier administration

You will receive the following items with your RAVICTI prescription (you can also ask Hyperion UCD Support Services for them):

  • Oral dosing syringe
  • AdaptaCap™ Bottle Adapter, which makes it easy for you to draw liquid from a bottle into an oral syringe

     

Directions for taking RAVICTI by mouth

     

Directions for taking RAVICTI if you have a nasogastric or gastrostomy feeding tube

AdaptaCap is a trademark of Baxter International Inc.

You can get samples of all of the dosing tools in the RAVICTI Patient Starter Kit.
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How safe is RAVICTI?

RAVICTI is safe and effective in adults and children 2 years of age and older. However, as with all medications, there are risks and benefits to taking RAVICTI. It is important to know some of the possible side effects of RAVICTI.

Who should not take RAVICTI?
  • Children less than 2 months of age should not take RAVICTI because it may not be digested in babies less than 2 months of age.
  • Do not take RAVICTI if you are allergic to phenylbutyrate.

Call your doctor or go to the nearest hospital emergency room if you get wheezing, shortness of breath, cough, low blood pressure, flushing, nausea, or a rash while taking RAVICTI.

If you or your child are allergic to phenylbutyrate, you or your child should not take RAVICTI.

RAVICTI may cause side effects.

The following are the most common side effects experienced with RAVICTI:

  • In adults: diarrhea, gas, headache, nausea, vomiting, tiredness, decreased appetite, high blood levels of ammoniaA waste product of protein digestion that is removed by the urea cycle process., and dizziness.
  • In children: upper abdomen (stomach) pain, nausea, vomiting, diarrhea, decreased appetite, high blood levels of ammonia, and headache.
RAVICTI may cause serious side effects

Phenylacetate, a breakdown product of RAVICTI, may cause nervous system side effects. Call your doctor or get medical help right away if you or your child get any of these symptoms while taking RAVICTI:

  • Sleepiness, weakness, lightheadedness, change in taste, problems with hearing, confusion, problems with memory, worsening neuropathy (numbness, tingling, or burning in your hands or feet), and/or headache.

Tell your doctor if you or your child experience any bothersome side effects that do not go away. Some of the side effects may be similar to symptoms experienced with high blood ammonia. You should always contact your doctor and/or go to the hospital if you suspect that you are having a hyperammonemic crisisIndicated by loss of appetite, vomiting, lethargy, and behavioral abnormalities and requires immediate medical attention.. The side effects listed above are not all of the possible side effects you could experience while on RAVICTI. See the Medication Guide (PDF) for more information.

Call your doctor for medical advice about side effects. You may report side effects to the FDA at 1-800-FDA-1088 (1-800-332-1088).

What to tell your doctor before taking RAVICTI

Medical conditions
Tell your doctor if you have liver or kidney problems, pancreas or bowel (intestine) problems, or any other medical conditions.

Pregnancy and nursing mothers
Tell your doctor if you are pregnant or plan to become pregnant. It is not known if RAVICTI will harm your unborn baby because tests have not been conducted. Tell your doctor if you are breastfeeding or plan to breastfeed. It is not known if RAVICTI passes into breast milk. You and your doctor should decide if you will take RAVICTI while breastfeeding.

Medicines
Tell your doctor about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and dietary or herbal supplements. It’s important to know the medicines you take, so keep a list to show your doctor and pharmacist when you are prescribed a new medicine.

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IMPORTANT SAFETY INFORMATIONRAVICTI® (GLYCEROL PHENYLBUTYRATE) ORAL LIQUID INDICATIONS AND USAGE

READ MORE

WHAT IS RAVICTI?

RAVICTI is a prescription medicine used in adults and children 2 years of age and older 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 treatment of acute hyperammonemia in people with UCDs.

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

It is not known if RAVICTI is safe and effective in children 2 months to less than 2 years of age.

RAVICTI IMPORTANT SAFETY INFORMATION

WHO SHOULD NOT TAKE RAVICTI:

  • Children less than 2 months of age should not take RAVICTI because it may not be digested in babies less than 2 months of age.
  • Do not take RAVICTI if you are allergic to phenylbutyrate. Call your doctor or go to the nearest hospital emergency room if you get wheezing, shortness of breath, cough, low blood pressure, flushing, nausea, or a rash while taking RAVICTI.
  • RAVICTI is not used for the treatment of acute hyperammonemia in people with UCDs.
  • It is not known if RAVICTI is safe and effective for the treatment of N-acetylglutamate synthase (NAGS) deficiency.
  • It is not known if RAVICTI is safe and effective in children 2 months to less than 2 years of age.

RAVICTI MAY CAUSE SERIOUS SIDE EFFECTS

Phenylacetate, a breakdown product of RAVICTI, may cause nervous system side effects. Call your doctor or get medical help right away if you get any of these symptoms while taking RAVICTI:

  • sleepiness
  • weakness
  • lightheadedness
  • change in taste
  • problems with hearing
  • confusion
  • problems with memory
  • worsening neuropathy (numbness, tingling, or burning in your hands or feet)
  • headache

WHAT ARE THE POSSIBLE SIDE EFFECTS OF RAVICTI?

The most common side effects of RAVICTI in adults include:

  • diarrhea
  • nausea
  • decreased appetite
  • gas
  • vomiting
  • high blood levels of ammonia
  • headache
  • tiredness
  • dizziness

The most common side effects of RAVICTI in children include:

  • upper abdomen (stomach) pain
  • diarrhea
  • high blood levels of ammonia
  • nausea
  • decreased appetite
  • headache
  • vomiting

Tell your doctor if you have any side effect that bothers you or that does not go away. These are not all of the possible side effects of RAVICTI. Call your doctor for medical advice about side effects.

BEFORE YOU TAKE RAVICTI, TELL YOUR DOCTOR IF YOU:

  • Have liver or kidney problems
  • Have pancreas or bowel (intestine) problems
  • Have any other medical conditions
  • Are pregnant or plan to become pregnant. It is not known if RAVICTI will harm your unborn baby.
  • Are breastfeeding or plan to breastfeed. It is not known if RAVICTI passes into your breast milk. RAVICTI may harm your baby, so you and your doctor should decide if you will take RAVICTI while breastfeeding.

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088. You may also contact the Hyperion Therapeutics Medical Information Department toll-free at 1-855-UCD-SUPT (1-855-823-7878) or drugsafety@hyperiontx.com.

For more information about RAVICTI, please see the full Prescribing Information (PDF) and Medication Guide (PDF) for RAVICTI.