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  • In Study 6 (right), mean venous ammonia levels in patients starting treatment at less than 2 months of age (n=16)b were less than 100 μmol/L during treatment with RAVICTI for up to 24 months (range, 35-94 μmol/L).1
  • In this study, 16 of 16 (100%) patients less than 2 months of age successfully transitioned to RAVICTI within 7 days (4 days of transition followed by 3 days of observation).1,c
  • Of 16 patients starting treatment at less than 2 months of age in this 24-month study, 5 patients (31%) reported a total of 7 hyperammonemic crises, all of which occurred in patients less than 1 month of age.1

RAVICTI provided ammonia control in pediatric patients less than 2 months of age with UCDs in clinical trial1,a

Graph showing efficacy of RAVICTI ammonia level control in UCD patients less than 2 months old, with mean normalized blood ammonia between 60 and 130 μmol/L at baseline and between 50 and 70 μmol/L at Month 24

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aAn uncontrolled, open-label study was conducted in 16 children less than 2 months of age to assess transition to RAVICTI over a period of 7 days, followed by monthly assessment of ammonia control and hyperammonemic crises over a 24-month period. Ammonia values across different laboratories were normalized to a common normal pediatric range of 28 to 57 μmol/L.1

bSixteen, 14, 12, 6, and 3 patients completed 1, 3, 6, 12, and 18 months of treatment, respectively (median exposure of 10 months [range, 2 to 20 months]).1

cSuccessful transition was defined as no signs and symptoms of hyperammonemia and a venous ammonia value less than 100 μmol/L.1

  • In Study 6 (right), mean venous ammonia levels in patients 2 months to less than 2 years of age (n=10)e were less than 100 μmol/L during treatment with RAVICTI for up to 6 months (range, 53-93 μmol/L).1
  • In Study 6, 9 of 10 (90%) patients 2 months to less than 2 years of age successfully transitioned to RAVICTI within 7 days.1,f
  • In Studies 4/4E and 5, mean venous ammonia levels in patients 2 months to less than 2 years of age (n=7)d were less than 100 μmol/L during treatment with RAVICTI for up to 12 months (range, 31-65 μmol/L).1
  • Of the 17 pediatric patients 2 months to less than 2 years of age in 3 open-label studies, 7 patients (41%) reported a total of 11 hyperammonemic crisis.1,g

RAVICTI provided ammonia control in pediatric patients 2 months to less than 2 years of age with UCDs in clinical trial1,d

Graph showing efficacy of RAVICTI ammonia level control in pediatric patients from 2 months to less than 2 years, with mean normalized blood ammonia between 80 and 130 μmol/L at baseline and between 50 and 70 μmol/L at Month 6

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dUncontrolled, open-label studies were conducted to assess monthly ammonia control and hyperammonemic crises with RAVICTI in pediatric patients 2 months to less than 2 years of age (study 4/4E, study 5, and study 6). Patients in study 5 previously participated in study 4/4E. A total of 17 pediatric patients with UCDs 2 months to less than 2 years of age participated in the studies. Ammonia values across different laboratories were normalized to a common normal pediatric range of 28 to 57 μmol/L.1

eNine, 7, 7, 4, 1 and 4 pediatric patients who completed 1, 3, 6, 12, 18 and 24 months, respectively (mean and median exposure of 9 and 9 months, respectively).1

fSuccessful transition was defined as no signs and symptoms of hyperammonemia and a venous ammonia value less than 100 μmol/L.1

gHyperammonemic crisis was defined as having signs and symptoms consistent with hyperammonemia (such as frequent vomiting, nausea, headache, lethargy, irritability, combativeness, and/or somnolence), associated with high ammonia levels and requiring medical intervention. High ammonia was defined as greater than 100 μmol/L, as demonstrated in study 6, and 93 μmol/L was the highest ammonia level reported in pediatric patients in an extension phase study.1

RAVICTI provided effective 24-hour ammonia control in pediatric patients with UCDs1,h

Graph showing efficacy of RAVICTI 24 hour ammonia level control in UCD pediatric patients from 2 to 17 compared to sodium phenylbutyrate, with RAVICTI mean normalized blood ammonia between 18 and 26 μmol/L pre-dose and between 15 and 22 μmol/L at Hour 24, lower than sodium phenylbutyrate

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  • In patients with UCDs between 2 and 5 years of age (n=11), the ammonia AUC0–24h value was 632 μmol⋅h/L vs 720 μmol⋅h/L on RAVICTI vs sodium phenylbutyrate (NaPBA), respectively.1
  • In patients 6 to 17 years of age (n=11), the ammonia AUC0–24h value was 604 μmol⋅h/L vs 815 μmol⋅h/L on RAVICTI vs NaPBA, respectively.1
  • No hyperammonemic crises were reported in the study during treatment with RAVICTI or sodium phenylbutyrate in pediatric patients ≥6 to <18 years.

hThe efficacy of RAVICTI in pediatric patients 2 to 17 years of age was evaluated in 2 fixed-sequence, open-label, NaPBA-to-RAVICTI crossover studies (studies 3 and 4). Study 3 was 7 days in duration and study 4 was 10 days in duration. These studies compared blood ammonia levels of patients on RAVICTI with venous ammonia levels of patients on NaPBA in 26 pediatric patients between 2 months and 17 years of age. The AUC0–24h for blood ammonia in 11 pediatric patients 6 to 17 years of age (study 3) and 11 pediatric patients 2 to 5 years of age (study 4) were similar between treatments.1

AUC, area under the curve.

  • Mean fasting venous ammonia values in pediatric patients (n=26) 6 to 17 years of age were within normal limits (≤35 μmol/L) during long-term treatment with RAVICTI (range, 17–23 μmol/L).1
  • Of the 26 pediatric patients 6 to 17 years of age participating in these 2 trials, 5 patients (19%) reported a total of 5 hyperammonemic crises.1

RAVICTI provided effective ammonia control sustained over 12 months in pediatric patients with UCDs1,h

Graph showing RAVICTI efficacy of sustained ammonia level control over 12 months in pediatric UCD patients, with mean normalized blood ammonia between 18 and 22 μmol/L at baseline and between 15 and 21 μmol/L at Month 12

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hLong-term (12-month), uncontrolled, open-label studies were conducted to assess monthly ammonia control and hyperammonemic crises. In 2 studies, a total of 26 pediatric patients 6 to 17 years of age were enrolled, and all but 1 had been converted from NaPBA to RAVICTI. Ammonia values across different laboratories were normalized to a common normal range of 9 to 35 μmol/L.1

RAVICTI provided consistent 24-hour ammonia levels in adult patients during a non-inferiority study compared with NaPB1,j

Graph showing efficacy of RAVICTI for 24 hour ammonia level control in adults with Urea Cycle Disorder compared to sodium phenylbutyrate, with RAVICTI mean normalized blood ammonia between 22 and 30 μmol/L pre-dose and at Hour 24, lower than sodium phenylbutyrate

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  • Mean 24-hour AUC0–24h values during steady-state dosing were 866 μmol x h/L and 977 μmol x h/L with RAVICTI and NaPBA, respectively (ratio of geometric means, 0.91 [95% CI 0.8, 1.04]).1
  • Among the 45 patients in this short-term study, no hyperammonemic crises were reported during treatment with RAVICTI.2

jA randomized, double-blind, active-controlled, crossover, noninferiority study compared RAVICTI with NaPBA by evaluating venous ammonia levels in patients with UCDs who had been on NaPBA prior to enrollment. Forty-five patients were randomized 1:1 to receive either NaPBA for 2 weeks then RAVICTI for 2 weeks, or RAVICTI for 2 weeks then NaPBA for 2 weeks. RAVICTI was noninferior to NaPBA with respect to the AUC0–24h for ammonia.1

AUC, area under the curve.

  • Mean fasting venous ammonia values in adults (n=51) were within normal limits (≤35 μmol/L) during long-term treatment with RAVICTI (range, 6–30 μmol/L).1
  • Of the 51 adult patients participating in the 12-month, open-label treatment with RAVICTI, 7 patients (14%) reported a total of 10 hyperammonemic crises.1

RAVICTI provided effective ammonia control sustained over 12 months in adult patients with UCDs1,k

Graph showing efficacy of RAVICTI in ammonia control sustained over 12 months; graph tracks Urea Cycle Disorder in adults, with mean normalized blood ammonia between 27 and 31 μmol/L at baseline and between 24 and 29 μmol/L at Month 12

Click to enlarge

Tap to enlarge

kA long-term (12-month), uncontrolled, open-label study was conducted to assess monthly ammonia control and hyperammonemic crises. A total of 51 adult patients were in the study and all but 6 had been converted from NaPBA to RAVICTI. Ammonia values across different laboratories were normalized to a common normal range of 9 to 35 μmol/L.1

Safety & Tolerability

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tolerability of RAVICTI.

Prescribing RAVICTI

Review the patient treatment profile and administration features of RAVICTI.

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References: 1. RAVICTI (glycerol phenylbutyrate) Oral Liquid [prescribing information] Amgen. 2. Longo N, Holt RJ. Glycerol phenylbutyrate for the maintenance treatment of patients with deficiencies in enzymes of the urea cycle. Exp Opin Orphan Drugs. 2017;5(12):999-1010.

INDICATION and IMPORTANT SAFETY INFORMATION

INDICATION

RAVICTI (glycerol phenylbutyrate) Oral Liquid is indicated for use as a nitrogen-binding agent for chronic management of patients with urea cycle disorders (UCDs) who cannot be managed by dietary protein restriction and/or supplementation alone. RAVICTI must be used with dietary protein restriction and, in some cases, dietary supplements (e.g. essential amino acids, arginine, citrulline, protein-free calorie supplements).

LIMITATIONS OF USE
  • RAVICTI is not indicated for the treatment of acute hyperammonemia in patients with UCDs because more rapidly acting interventions are essential to reduce plasma ammonia levels.
  • The safety and efficacy of RAVICTI for the treatment of N-acetylglutamate synthase (NAGS) deficiency has not been established.

IMPORTANT SAFETY INFORMATION

CONTRAINDICATIONS
  • Patients with known hypersensitivity to phenylbutyrate: Reactions include wheezing, dyspnea, coughing, hypotension, flushing, nausea, and rash.
WARNINGS AND PRECAUTIONS
  • Neurotoxicity: Phenylacetate (PAA), the major metabolite of RAVICTI, may be toxic at levels of 500 micrograms/mL or greater. If symptoms of vomiting, nausea, headache, somnolence, or confusion, are present in the absence of high ammonia or other intercurrent illness which explains these symptoms, consider the potential for PAA neurotoxicity which may need reduction in the RAVICTI dosage.
  • Pancreatic Insufficiency or Intestinal Malabsorption: Low or absent pancreatic enzymes or intestinal disease resulting in fat malabsorption may result in reduced or absent digestion of RAVICTI and/or absorption of phenylbutyrate and reduced control of plasma ammonia. Monitor ammonia levels closely.
ADVERSE REACTIONS

The most common adverse reactions reported in clinical trials (at least 10% of patients) were:

  • Adult patients: diarrhea, flatulence, and headache occurred during 4-week treatment (n=45) with RAVICTI; nausea, vomiting, diarrhea, decreased appetite, dizziness, headache, and fatigue occurred during 12-month treatment (n=51) with RAVICTI.
  • Pediatric patients ages 2 to 17 years: upper abdominal pain, rash, nausea, vomiting, diarrhea, decreased appetite, and headache occurred during 12-month treatment (n=26) with RAVICTI.
  • Pediatric patients ages 2 months to less than 2 years: neutropenia, vomiting, constipation, diarrhea, pyrexia, hypophagia, cough, nasal congestion, rhinorrhea, rash, and papule occurred during 12-month treatment (n=17) with RAVICTI.
  • Pediatric patients less than 2 months of age: vomiting, rash, gastroesophageal reflux, increased hepatic enzymes, feeding disorder (decreased appetite, hypophagia), anemia, cough, dehydration, metabolic acidosis, thrombocytosis, thrombocytopenia, neutropenia, lymphocytosis, diarrhea, flatulence, constipation, pyrexia, lethargy, and irritability/agitation occurred during 24-month treatment (n=16) with RAVICTI.
DRUG INTERACTIONS
  • Corticosteroids, valproic acid, or haloperidol may increase plasma ammonia level. Monitor ammonia levels closely.
  • Probenecid may affect renal excretion of metabolites of RAVICTI, including phenylacetylglutamine (PAGN) and PAA.
  • CYP3A4 substrates with narrow therapeutic index (eg, alfentanil, quinidine, cyclosporine): RAVICTI may decrease exposure to the concomitant drug.
  • Midazolam: Use of RAVICTI decreased exposure of midazolam with concomitant use.
USE IN SPECIFIC POPULATIONS
  • Pregnancy: RAVICTI should be used with caution in patients who are pregnant or planning to become pregnant. Based on animal data, RAVICTI may cause fetal harm. Report pregnancies to Amgen at 1‐866‐479‐6742.
  • Lactation: breastfeeding is not recommended during treatment with RAVICTI. There are no data on the presence of RAVICTI in human milk, the effects on the breastfed infant, nor the effects on milk production.

Please see Full Prescribing Information.

Use and 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) 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:

  • 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, click here for the Medication Guide and discuss with your doctor.

Use and Important Safety Information

What is the most important safety information I should know about BUPHENYL?
 BUPHENYL may cause serious side effects, including:
Nervous system side effects (Neurotoxicity) Phenylacetate (PAA), a breakdown product of BUPHENYL, may cause nervous system side effects. Call your doctor or get medical help right away if you have any of these symptoms while taking BUPHENYL:

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

What is 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 only treats high blood levels of ammonia in patients with carbamylphosphate synthetase (CPS), ornithine transcarbamylase (OTC) and argininosuccinic acid synthetase (AS) deficiencies.
  • BUPHENYL can be used in infants up to 28 days old who have a complete enzyme deficiency, and in patients 1 month of age and older who have a partial enzyme deficiency and have a history of brain damage from high blood levels of ammonia.
  • BUPHENYL must be used along with a low-protein diet and in some cases, dietary supplements.
  • BUPHENYL se debe usar junto con una dieta baja en proteínas y, en algunos casos, suplementos dietarios.
  • BUPHENYL is not used to treat acute (severe) hyperammonemia, which is a medical emergency.

Do not take BUPHENYL if you are allergic to phenylbutyrate. Call your doctor or go to the nearest hospital emergency room if you experience an allergic reaction while taking BUPHENYL.

Before taking BUPHENYL, tell your doctor about all of your medical conditions, including 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.
  • are pregnant or plan to become pregnant. It is not known if BUPHENYL will harm your unborn baby.
  • are breastfeeding or plan to breastfeed. It is not known if BUPHENYL passes into your breast milk. Breastfeeding is not recommended during treatment with BUPHENYL. Talk to your doctor about the best way to feed your baby if you take BUPHENYL.

What are possible side effects of BUPHENYL?

BUPHENYL may cause serious side effects, including:

  • See “What is the most important information I should know about BUPHENYL?”

The most common side effects of BUPHENYL include:

  • decreased appetite
  • body odor
  • bad taste
  • absent or irregular periods in women

These are not all of the possible side effects of BUPHENYL. 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, click here for the Patient Package Insert and discuss with your doctor.

INDICATION and IMPORTANT SAFETY INFORMATION

INDICATION

RAVICTI (glycerol phenylbutyrate) Oral Liquid is indicated for use as a nitrogen-binding agent for chronic management of patients with urea cycle disorders (UCDs) who cannot be managed by dietary protein restriction and/or supplementation alone. RAVICTI must be used with dietary protein restriction and, in some cases, dietary supplements (e.g. essential amino acids, arginine, citrulline, protein-free calorie supplements).

INDICATION

RAVICTI (glycerol phenylbutyrate) Oral Liquid is indicated for use as a nitrogen-binding agent for chronic management of patients with urea cycle disorders (UCDs) who cannot be managed by dietary protein restriction and/or supplementation alone. RAVICTI must be used with dietary protein restriction and, in some cases, dietary supplements (e.g. essential amino acids, arginine, citrulline, protein-free calorie supplements).

LIMITATIONS OF USE
  • RAVICTI is not indicated for the treatment of acute hyperammonemia in patients with UCDs because more rapidly acting interventions are essential to reduce plasma ammonia levels.
  • The safety and efficacy of RAVICTI for the treatment of N-acetylglutamate synthase (NAGS) deficiency has not been established.

IMPORTANT SAFETY INFORMATION

CONTRAINDICATIONS
  • Patients with known hypersensitivity to phenylbutyrate: Reactions include wheezing, dyspnea, coughing, hypotension, flushing, nausea, and rash.
WARNINGS AND PRECAUTIONS
  • Neurotoxicity: Phenylacetate (PAA), the major metabolite of RAVICTI, may be toxic at levels of 500 micrograms/mL or greater. If symptoms of vomiting, nausea, headache, somnolence, or confusion, are present in the absence of high ammonia or other intercurrent illness which explains these symptoms, consider the potential for PAA neurotoxicity which may need reduction in the RAVICTI dosage.
  • Pancreatic Insufficiency or Intestinal Malabsorption: Low or absent pancreatic enzymes or intestinal disease resulting in fat malabsorption may result in reduced or absent digestion of RAVICTI and/or absorption of phenylbutyrate and reduced control of plasma ammonia. Monitor ammonia levels closely.
ADVERSE REACTIONS

The most common adverse reactions reported in clinical trials (at least 10% of patients) were:

  • Adult patients: diarrhea, flatulence, and headache occurred during 4-week treatment (n=45) with RAVICTI; nausea, vomiting, diarrhea, decreased appetite, dizziness, headache, and fatigue occurred during 12-month treatment (n=51) with RAVICTI.
  • Pediatric patients ages 2 to 17 years: upper abdominal pain, rash, nausea, vomiting, diarrhea, decreased appetite, and headache occurred during 12-month treatment (n=26) with RAVICTI.
  • Pediatric patients ages 2 months to less than 2 years: neutropenia, vomiting, constipation, diarrhea, pyrexia, hypophagia, cough, nasal congestion, rhinorrhea, rash, and papule occurred during 12-month treatment (n=17) with RAVICTI.
  • Pediatric patients less than 2 months of age: vomiting, rash, gastroesophageal reflux, increased hepatic enzymes, feeding disorder (decreased appetite, hypophagia), anemia, cough, dehydration, metabolic acidosis, thrombocytosis, thrombocytopenia, neutropenia, lymphocytosis, diarrhea, flatulence, constipation, pyrexia, lethargy, and irritability/agitation occurred during 24-month treatment (n=16) with RAVICTI.
DRUG INTERACTIONS
  • Corticosteroids, valproic acid, or haloperidol may increase plasma ammonia level. Monitor ammonia levels closely.
  • Probenecid may affect renal excretion of metabolites of RAVICTI, including phenylacetylglutamine (PAGN) and PAA.
  • CYP3A4 substrates with narrow therapeutic index (eg, alfentanil, quinidine, cyclosporine): RAVICTI may decrease exposure to the concomitant drug.
  • Midazolam: Use of RAVICTI decreased exposure of midazolam with concomitant use.
USE IN SPECIFIC POPULATIONS
  • Pregnancy: RAVICTI should be used with caution in patients who are pregnant or planning to become pregnant. Based on animal data, RAVICTI may cause fetal harm. Report pregnancies to Amgen at 1‐866‐479‐6742.
  • Lactation: breastfeeding is not recommended during treatment with RAVICTI. There are no data on the presence of RAVICTI in human milk, the effects on the breastfed infant, nor the effects on milk production.

Please see Full Prescribing Information.

Use and 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) 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:

  • 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, click here for the Medication Guide and discuss with your doctor.