Learn about RAVICTI® (glycerol phenylbutyrate) Oral Liquid

If you or a loved one is living with a urea cycleA metabolic process in which waste (nitrogen) from the breakdown of proteins in the body is changed by the liver into urea, which is excreted in the urine. 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.1

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.1 It is the only FDA-approved oral liquid medicine for UCDs that2:

  • Can be taken by mouth1
  • Has little-to-no taste and is odorless3
  • Along with a low-proteinEssential to all living cells, simplified by body processes to simple amino acids. diet, can keep your ammonia at safe levels1

RAVICTI should only be used 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 must be used along with a low-protein diet and, in some cases, dietary supplements.1

RAVICTI can be used in people with different UCDs, except N-acetylglutamate synthase (NAGS) deficiency1

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 RAVICTI1,4
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-hyperammonemiaElevated levels of ammonia in the blood.-homocitrullinuria (HHH) syndrome
Citrulinemia (aspartate glutamate translocase)

RAVICTI is not used for the treatment of acute hyperammonemia in people with UCDs.1

It is not known if RAVICTI is safe and effective for the treatment of N-acetylglutamate synthase (NAGS) deficiency.1

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

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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.5-7

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.5-11 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.

Selected Important Risk Information
RAVICTI is not indicated for the treatment of acute hyperammonemia in patients with UCDs as other treatments are used to quickly lower ammonia levels.1

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 levels within the normal range at 24 hours.1

  • Three short-term studies (ranging from 7 days to 4 weeks) compared ammonia levels in people with UCDs receiving RAVICTI or sodium phenylbutyrate.. Read the Important Safety Information and Full Prescribing Information for BUPHENYL® (sodium phenylbutyrate) Tablets or Powder.
  • No hyperammonemic crises were reported with RAVICTI in these short-term studies in adults and children with UCDs.

 

On RAVICTI, average blood ammonia levels stayed within a normal range during the treatment year.1

  • In adults, average blood ammonia levels were within normal limits during 12 months of treatment with RAVICTI (range: 6-30 µmol/L). This range was considered normal during the treatment year.1
    • 7 out of 51 adults (14%) in the study reported a total of 10 hyperammonemic crises1
  • 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.1
    • 5 out of 26 children 6 to 17 years of age (19%) in the study reported a total of 5 hyperammonemic crises.1
    • Two long-term studies (12 months) compared monthly ammonia levels in people with UCDs receiving RAVICTI.1

After using RAVICTI for 2 weeks in clinical studies, patients and caregivers were asked if they wanted to continue using RAVICTI for up to a year in an additional study:

    • 91% of adults and their caregivers (40 of 44) chose to continue with RAVICTI10
    • 100% of children 6 to 17 years of age and their caregivers (26 of 26) chose to continue with RAVICTI 9

Based on your ammonia level, you could experience varying symptoms and consequences.
Read more »

How Does RAVICTI work?

RAVICTI is the only ammonia-removing medicine available as an oral liquid1

RAVICTI is an oral liquid prescription medicine that1:

  • Can be taken by mouth1
  • Has little-to-no taste and is odorless3
  • Does not have pills or powder that require any preparation1

RAVICTI is slowly released throughout the day1

The active ingredient in RAVICTI is released slowly in your body, mostly in the small intestine. This means RAVICTI remains active in your body removing ammoniaA waste product of protein digestion that is removed by the urea cycle process. during the day. 1

RAVICTI has been studied over a 12-month 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.1

Selected Important Safety Information
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.

How do I take RAVICTI?

RAVICTI is easy to take: 3 times daily with meals

RAVICTI is an oral liquid prescription medicine that1:

  • Can be taken by mouth1
  • Has little-to-no taste and is odorless3
  • Does not have pills or powder that require preparation1

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.1

The maximum approved daily dose of RAVICTI is 17.5 mL for both adults and children.1

RAVICTI comes with dosing tools for easy administration

 

You will receive the following items with your RAVICTI prescription (you can also ask Horizon 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

Administering RAVICTI orally

Administering RAVICTI with nasogastric or gastrostomy feeding tube

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?1
  • 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 serious side effects1

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.1

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, high blood levels of ammonia, and dizziness.
  • The most common side effects of RAVICTI in children include upper abdomen (stomach) pain, nausea, vomiting, diarrhea, decreased appetite, high blood levels of ammonia, and 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.1

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

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.

Medicines
Tell your doctor about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and dietary or herbal supplementsEssential amino acids like arginine or citrulline may be added depending on the urea cycle defect.. 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

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Approved Uses for RAVICTI® (glycerol phenylbutyrate) Oral Liquid

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 only 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.

Detailed 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 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 experience 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), or headache.

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, high blood levels of ammonia, and dizziness.

The most common side effects of RAVICTI in children include upper abdomen (stomach) pain, nausea, vomiting, diarrhea, decreased appetite, high blood levels of ammonia, and headache.

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

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.

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

References:  1. RAVICTI [package insert]. Deerfield, IL: Horizon Pharma USA, Inc.; 2015.  2. US Food and Drug Administration. Orange book: approved drug products with therapeutic equivalence evaluations. http://www.accessdata.fda.gov/scripts/cder/ob/docs/tempai.cfm. Updated May 17, 2013. Accessed July 1, 2015.  3. Diaz GA, Krivitzky LS, Mokhtarani M, et al. Ammonia control and neurocognitive outcome among urea cycle disorder patients treated with glycerol phenylbutyrate. Hepatology. 2013;57(6):2171-2179. doi:10.1002/hep.26058.  4. Lanpher BC, Gropman A, Chapman KA, Lichter-Konecki U, Summar ML; Urea Cycle Disorders Consortium. Urea cycle disorders overview. In: Pagon RA, Bird TD, Dolan CR, Stephen K, eds. GeneReviews. Seattle, WA: University of Washington, Seattle; 2011. http://www.ncbi.nlm.nih.gov/books/NBK1217/. Published April 29, 2003. Updated September 1, 2011. Accessed November 26, 2012.  5. Summar M, Tuchman M. Proceedings of a consensus conference for the management of patients with urea cycle disorders. J Pediatr. 2001;138(1)(suppl):S6-S10. doi:10.1067/mpd.2001.111831.  6. Gropman A. Brain imaging in urea cycle disorders. Mol Genet Metab. 2010;100(suppl 1):S20-S30. doi:10.1016/j.ymgme.2010.01.017.  7. Batshaw ML, MacArthur RB, Tuchman M. Alternative pathway therapy for urea cycle disorders: twenty years later. J Pediatr. 2001;138(suppl 1):S46-S55. doi:10.1067/mpd.2001.111836.  8. Gropman AL, Summar M, Leonard JV. Neurological implications of urea cycle disorders. J Inherit Metab Dis. 2007;30(6):865-869. doi:10.1007/s10545-007-0709-5.  9. Msall M, Batshaw ML, Suss R, Brusilow SW, Mellits ED. Neurologic outcome in children with inborn errors of urea synthesis: outcome of urea-cycle enzymopathies. N Engl J Med. 1984;310(23):1500-1505. doi:10.1056/nejm198406073102304.  10. Msall M, Monahan PS, Chapanis N, Batshaw ML. Cognitive development in children with inborn errors of urea synthesis. Acta Paediatr Jpn. 1988;30(4):435-441. doi:10.1111/j.1442-200x.1988.tb02534.x.  11. Uchino T, Endo F, Matsuda I. Neurodevelopmental outcome of long-term therapy of urea cycle disorders in Japan. J Inherit Metab Dis. 1998;21(suppl 1):151-159.

References:  1. RAVICTI [package insert]. Deerfield, IL: Horizon Pharma USA, Inc.; 2015.  2. US Food and Drug Administration. Orange book: approved drug products with therapeutic equivalence evaluations. http://www.accessdata.fda.gov/scripts/cder/ob/docs/tempai.cfm. Updated May 17, 2013. Accessed July 1, 2015.  3. Lanpher BC, Gropman A, Chapman KA, Lichter-Konecki U, Summar ML; Urea Cycle Disorders Consortium. Urea cycle disorders overview. In: Pagon RA, Bird TD, Dolan CR, Stephen K, eds. GeneReviews. Seattle, WA: University of Washington, Seattle; 2011. http://www.ncbi.nlm.nih.gov/books/NBK1217/. Published April 29, 2003. Updated September 1, 2011. Accessed November 26, 2012.  4. Diaz GA, Krivitzky LS, Mokhtarani M, et al. Ammonia control and neurocognitive outcome among urea cycle disorder patients treated with glycerol phenylbutyrate. Hepatology. 2013;57(6):2171-2179. doi:10.1002/hep.26058.

References:  1. Hook DG. Preventing protein catabolism. Pasadena, CA: National Urea Cycle Disorders Foundation; 1997.  2. Häberle J. Clinical practice: the management of hyperammonemia. Eur J Pediatr. 2011;170(1):21-34. doi:10.1007/s00431-010-1369-2.  3. RAVICTI [package insert]. Deerfield, IL: Horizon Pharma USA, Inc.; 2015.  4. Gropman AL, Summar M, Leonard JV. Neurological implications of urea cycle disorders. J Inherit Metab Dis. 2007;30(6):865-869. doi:10.1007/s10545-007-0709-5.  5. Brusilow SW, Maestri NE. Urea cycle disorders: diagnosis, pathophysiology, and therapy. Adv Pediatr. 1996;43:127-170.  6. Häberle J, Boddaert N, Burlina A, et al. Suggested guidelines for the diagnosis and management of urea cycle disorders. Orphanet J Rare Dis. 2012;7:32. doi:10.1186/1750-1172-7-32.  7. Lanpher BC, Gropman A, Chapman KA, Lichter-Konecki U, Summar ML; Urea Cycle Disorders Consortium. Urea cycle disorders overview. In: Pagon RA, Bird TD, Dolan CR, Stephen K, eds. GeneReviews. Seattle, WA: University of Washington, Seattle; 2011. http://www.ncbi.nlm.nih.gov/books/NBK1217/. Published April 29, 2003. Updated September 1, 2011. Accessed November 26, 2012.  8. Ah Mew N, Krivitzky L, McCarter R, Batshaw M, Tuchman M; for Urea Cycle Disorders Consortium of the Rare Diseases Clinical Research Network. Clinical outcomes of neonatal onset proximal versus distal urea cycle disorders do not differ. J Pediatr. 2013;162(2):324-329.e1. doi:10.1016/j.jpeds.2012.06.065.  9. Msall M, Batshaw ML, Suss R, Brusilow SW, Mellits ED. Neurologic outcome in children with inborn errors of urea synthesis: outcome of urea-cycle enzymopathies. N Engl J Med. 1984;310(23):1500-1505. doi:10.1056/nejm198406073102304.  10. Msall M, Monahan PS, Chapanis N, Batshaw ML. Cognitive development in children with inborn errors of urea synthesis. Acta Paediatr Jpn. 1988;30(4):435-441. doi:10.1111/j.1442-200x.1988.tb02534.x.  11. Summar M, Tuchman M. Proceedings of a consensus conference for the management of patients with urea cycle disorders. J Pediatr. 2001;138(1)(suppl):S6-S10. doi:10.1067/mpd.2001.111831.  12. Gropman A. Brain imaging in urea cycle disorders. Mol Genet Metab. 2010;100(suppl 1):S20-S30. doi:10.1016/j.ymgme.2010.01.017.  13. Uchino T, Endo F, Matsuda I. Neurodevelopmental outcome of long-term therapy of urea cycle disorders in Japan. J Inherit Metab Dis. 1998;21(suppl 1):151-159.  14. Batshaw ML, MacArthur RB, Tuchman M. Alternative pathway therapy for urea cycle disorders: twenty years later. J Pediatr. 2001;138(suppl 1):S46-S55. doi:10.1067/mpd.2001.111836.

References:  1. Lanpher BC, Gropman A, Chapman KA, Lichter-Konecki U, Summar ML; Urea Cycle Disorders Consortium. Urea cycle disorders overview. In: Pagon RA, Bird TD, Dolan CR, Stephen K, eds. GeneReviews. Seattle, WA: University of Washington, Seattle; 2011. http://www.ncbi.nlm.nih.gov/books/NBK1217/. Published April 29, 2003. Updated September 1, 2011. Accessed November 26, 2012.  2. Gropman AL, Summar M, Leonard JV. Neurological implications of urea cycle disorders. J Inherit Metab Dis. 2007;30(6):865-869. doi:10.1007/s10545-007-0709-5.  3. Brusilow SW, Maestri NE. Urea cycle disorders: diagnosis, pathophysiology, and therapy. Adv Pediatr. 1996;43:127-170.  4. Cederbaum S. What is a UCD? National Urea Cycle Disorders Foundation website. http://nucdf.org/ucd_kinds.htm. Accessed July 22, 2015.  5. RAVICTI [package insert]. Deerfield, IL: Horizon Pharma USA, Inc.; 2015. 

References:  1. Ah Mew N, Lanpher BC, Gropman A, Chapman KA, Simpson KL, Summar ML; Urea Cycle Disorders Consortium. Urea Cycle Disorders Overview. In: Pagon RA, Adam MP, Ardinger HH, et al, eds. GeneReviews. Seattle, WA: University of Washington, Seattle; 2015. http://www.ncbi.nlm.nih.gov/books/NBK1217/. Published April 29, 2003. Updated April 9, 2015. Accessed July 1, 2015.  2. Cederbaum S. What is a UCD? National Urea Cycle Disorders Foundation website. http://nucdf.org/ucd_kinds.htm. Accessed July 22, 2015.  3. US Food and Drug Administration. Medical foods guidance documents & regulatory information. http://www.fda.gov/food/guidanceregulation/guidancedocumentsregulatoryinformation/medicalfoods/default.htm. Updated November 26, 2014. Accessed July 22, 2015.  4. MedlinePlus. Nasogastric feeding tube. http://www.nlm.nih.gov/medlineplus/ency/patientinstructions/000182.htm. Updated December 2, 2014. Accessed July 22, 2015.  5. Kober S. The evolution of specialty pharmacy. Biotechnol Healthc. 2008;5(2):50-51.