Safety & Tolerability
Learn about the safety and
tolerability of RAVICTI.
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
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 umol/L, as demonstrated in study 6, and 93 umol/L is considered the upper limit of normal ammonia levels for pediatric patients without a UCD according to the US Prescribing Information.1
gThe 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
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
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.
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
Learn about the safety and
tolerability of RAVICTI.
Review the patient treatment profile and
administration features of RAVICTI.
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References: 1. RAVICTI (glycerol phenylbutyrate) Oral Liquid [prescribing information] Horizon. 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.
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).
The most common adverse reactions reported in clinical trials (at least 10% of patients) were:
Please see Full Prescribing Information.
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).
The most common adverse reactions reported in clinical trials (at least 10% of patients) were:
Please see Full Prescribing Information.