Common questions about RAVICTI for treating urea cycle disorders

Here are answers to common questions that you may have about RAVICTI® (glycerol phenylbutyrate).

What is RAVICTI?

RAVICTI is the only FDA-approved oral liquid medicine that can be used in adults and children aged 2 years and older for the long-term management of elevated levels of ammonia caused by a condition called a urea cycle disorder (UCD). RAVICTI should be used only if the UCD cannot be managed with a low-protein diet and dietary supplements alone.

RAVICTI is not used for the treatment of acute hyperammonemia.

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

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

Children younger than 2 months should not take RAVICTI because they may not be able to digest it.

Do not take RAVICTI if you are allergic to phenylbutyrate.

Can I take RAVICTI if I am pregnant or nursing?

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 your breast milk. RAVICTI may harm your baby, so you and your doctor should decide if you will take RAVICTI or breastfeed.

Patients and their caregivers should be informed that a registry for urea cycle disorder (UCD) patients has been established by Horizon Therapeutics in order to better assess long-term outcomes in patients with UCDs, including growth and neurocognitive outcomes and the outcome of pregnancy for women with UCDs who become pregnant. Patients and their caregivers are encouraged to participate in the registry and advised that their participation is voluntary. For more information regarding the registry program, visit www.ucdregistry.com or call 1-855-823-2595.

How do I know if RAVICTI is working?

Your doctor or nurse will draw blood frequently to measure your ammonia levels to find out if they are at a safe and normal level. This is to make sure RAVICTI works as well as possible. It is important to always take each dose exactly as your doctor prescribes. Missing a dose or taking the wrong amount may mean that your ammonia levels are not being well controlled.

What if I am switching to RAVICTI from BUPHENYL Tablets or Powder?

Read the Important Safety Information regarding BUPHENYL.

If your doctor switched you to RAVICTI, you should know that there are some differences from sodium phenylbutyrate. RAVICTI is a liquid that you can take by mouth. It has little to no taste, and there are no pills or powder to prepare. Your doctor may tell you to take an amount of RAVICTI that is different than the amount of sodium phenylbutyrate you were taking. Always take RAVICTI exactly as your doctor prescribes.

What should I do if I am taking other prescription medications in addition to RAVICTI?

Tell your doctor about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and dietary and 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.

What if I am having problems paying for RAVICTI?

If you qualify, Horizon UCD Support Services can help:

  • Support you with the healthcare coverage process
  • Pay for part of your copay or coinsurance
  • Potentially get you the medication at little or no cost

Important Safety information

Ravicti® (glycerol phenylbutyrate) oral liquid indications and usage

Approved Uses for RAVICTI® (glycerol phenylbutyrate) Oral Liquid

RAVICTI is a prescription medicine used in adults and children 2 months of age and older for long-term management of high levels of ammonia in the blood (hyperammonemia) caused by a condition called a urea cycle disorder (UCD). RAVICTI must be used along with a low-protein diet and, in some cases, dietary supplements. RAVICTI should only be used if the UCD cannot be managed with a low-protein diet and dietary supplements alone.

RAVICTI is not used to treat extremely high levels of ammonia in the blood (hyperammonemic crises) in people with UCDs as they may require rapidly acting medication.

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 for children younger than 2 months.

Detailed Important Safety Information

Who should not take RAVICTI:

Children younger than 2 months should not take RAVICTI because they may not be able to digest it.

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

RAVICTI may cause serious side effects:

The breakdown of RAVICTI produces the byproduct phenylacetate, 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, tiredness; nausea; or vomiting.

What are the possible side effects of RAVICTI?

The most common side effects of RAVICTI in adults include diarrhea, gas, headache, nausea, vomiting, tiredness, decreased appetite, and dizziness.

The most common side effects of RAVICTI in children ages 2 to 17 years include stomach pain, nausea, vomiting, diarrhea, decreased appetite, and headache.

The most common side effects of RAVICTI in children ages 2 months to younger than 2 years include decreased level of a type of white blood cell, vomiting, diarrhea, fever, decreased appetite, cough, nasal congestion, runny nose, and rash.

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, pancreas or bowel (intestine) problems, or any other medical conditions. Tell your doctor if you are pregnant or plan to become pregnant. It is not known if RAVICTI will harm your unborn baby. Tell your doctor if you 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.

Talk to your doctor about participating in a UCD registry. The purpose of this registry is to collect information about people with UCDs to improve care. For more information about the registry program, call 1-855-823-2595 or visit www.ucdregistry.com.

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.

This information is not comprehensive. To learn more, talk to your healthcare provider or pharmacist. The FDA-approved product labeling, including the Medication Guide can be found at Ravicti.com.

important safety information

buphenyl®(sodium phenylbutyrate) tablets and powder indications and usage

Approved Uses for BUPHENYL®

BUPHENYL (sodium phenylbutyrate) Tablets is a prescription medicine that can be taken by mouth and BUPHENYL (sodium phenylbutyrate) Powder is a prescription medicine that can be taken by mouth or feeding tube for the long-term management of high blood levels of ammonia (hyperammonemia) caused by a condition called a urea cycle disorder (UCD). BUPHENYL should be used if the UCD cannot be managed with a low-protein diet and dietary supplements alone.

BUPHENYL only treats high blood levels of ammonia in patients with the following enzyme deficiencies:

  • Carbamylphosphate synthetase (CPS)
  • Ornithine transcarbamylase (OTC)
  • Argininosuccinic acid synthetase (AS)

BUPHENYL can be used in infants up to 28 days old who have a complete enzyme deficiency (an enzyme in the urea cycle that does not work at all). It can also be used in people 1 month of age and up who have a partial enzyme deficiency (an enzyme in the urea cycle that only works partially) and have a history of brain damage from high blood levels of ammonia (hyperammonemia). It is important to have a healthcare provider diagnose this condition and prescribe a medication as early as possible to improve chance of survival.

BUPHENYL must be used along with a low-protein diet and, in some cases, dietary supplements.

Any episode related to acute hyperammonemia should be treated as a life-threatening emergency.

Important Safety Information (ISI)

Do not take BUPHENYL if you are allergic to phenylbutyrate, or for the treatment of acute hyperammonemia in people with UCDs.

Use of BUPHENYL may cause serious side effects to the nervous system due to phenylacetate, a breakdown product of BUPHENYL. Call your doctor or get medical help right away if you experience any of the following symptoms while taking BUPHENYL: sleepiness, weakness, lightheadedness, problems with memory, worsening neuropathy (numbness, tingling, or burning in your hands or feet), change in taste, problems with hearing, confusion, and headache.

Talk to your doctor before taking BUPHENYL if you have heart failure or decreased kidney function, which may lead to retention of the sodium content of BUPHENYL with potentially serious consequences such as worsening heart failure, high blood pressure, and swelling. You and your doctor should decide if you will take BUPHENYL if you have these medical conditions. Do not take BUPHENYL if you have liver or kidney problems, have any other medical conditions, if your child is 20kg or less, or if you are planning to become pregnant or breastfeed, as it is unknown if BUPHENYL will harm your unborn baby or will pass into your breastmilk.

The most common side effects of BUPHENYL include absent or irregular periods in women, decreased appetite, body odor, and bad taste.

The most common side effects of BUPHENYL® seen in a laboratory setting include changes to blood pH and electrolyte levels (such as chloride and phosphate), low protein levels in the blood, high levels of certain bone and liver enzymes (such as alkaline phosphatase and transaminases), and decreased red and white blood cell and platelet count.

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.

This information is not intended to replace discussions with your doctor. For additional information about BUPHENYL®, please consult the Full Prescribing Information and the Information for the Patient/Caregiver and talk to your doctor. BUPHENYL® is available by prescription only.

Please see the full Prescribing Information and Patient Package Insert for BUPHENYL.