How to Get RAVICTI

Partner With Your Doctor

Once you find out that you or your child has a urea cycle disorder (UCD), you need to learn everything you can about UCDs. Ask your doctor to explain things you don’t understand. If your doctor orders a test, find out why. Make sure you know what treatments are available. A doctor who treats you as a partner will welcome your questions. This will help you get the care you need. Create your Doctor Discussion Guide to help you prepare for your next visit.

Advocating for your child

See how parents learned to seek out information and advocate for their children in the healthcare system.

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Advocating for your child with a UCD
Read transcript

Amy:

It took me a few times, but now I’m that hospital mom, "Momma Bear."" After the lab techs come up, and they poke her twice, I’m the mom that’s not afraid to ask for the anesthesiologist because if they waste 6 different poke sites, just trying to start one IV, we're gonna be in trouble for that whole hospitalization.

And I had had different experiences with doctors and hospitals, so I knew right away that if you walk in and you act like you own the meeting, you own the meeting. And that’s what I did.

You have to be aware of what’s going on. And you know your child best.

David:

Thank God for doctors.

They’re trying to do their best to serve you. You have to treat them well. But at the same time, you have to be an advocate when you present to a emergency room or a medical situation with your child.

You won't get it from yelling. Don’t do anything like that. Just persistently be an advocate for your child.

You have to do it as a parent, and we’ve been teaching our son, to be his own advocate.

Do it sternly, firmly, consistently. Do not back down. If you do not get results, find somewhere where you can.

Filling your prescription for RAVICTI

Medicines like RAVICTI® (glycerol phenylbutyrate) Oral Liquid that treat rare or serious conditions are called specialty medicines. These medicines may need to be handled carefully or taken in a certain way. Usually, you can only get these medicines at a specialty pharmacy. When your doctor prescribes RAVICTI, it will come to you from a specialty pharmacy.

What is a specialty pharmacy?

Once you and your doctor choose RAVICTI, you will work with a specialty pharmacy to get the medicine. Unlike a local retail pharmacy, a specialty pharmacy takes care of getting your medicine from start to finish. Your specialty pharmacy will get your prescription from your doctor and send RAVICTI straight to your house or doctor's office. Your Patient Access Manager (PAM) can help answer questions during this process.

Horizon Patient Services is here to help

Horizon Patient Services, is dedicated to improve lives of people living with urea cycle disorders. The Horizon Patient Services program provides ongoing individualized support and education for you and your family. Your Patient Access Manager (PAM) leads your dedicated Horizon Patient Services team that is here to help you, from answering questions as your start taking RAVICTI® and through every step along the way.

Help is just a phone call away. To talk to a Patient Access Manager, call 1-833-830-RARE (1-833-830-7273).

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APPROVED USES and IMPORTANT SAFETY INFORMATION for RAVICTI
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.

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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 to treat extremely high levels of ammonia in the blood (hyperammonemic crisis) in people with UCDs.

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

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.

For additional important safety information, click here for the Full Prescribing Information and Medication Guide and discuss with your doctor.

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.

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 to treat extremely high levels of ammonia in the blood (hyperammonemic crisis) in people with UCDs.

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

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.

For additional important safety information, click here for the Full Prescribing Information and Medication Guide and discuss with your doctor.

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.