About RAVICTI
Review efficacy data for adult
and pediatric patients
The lifelong, chronic nature of UCDs means patients are constantly living on the edge1,2
A UCD is a rare genetic condition caused by an enzyme or transporter in the urea cycle that is missing or defective. The resulting dysfunction of the urea cycle may cause a buildup of ammonia in the blood.
Elevated ammonia levels (greater than or equal to 35 μmol/L), whether acute or chronic, may lead to serious medical consequences.4,5
Symptoms of elevated ammonia in patients with partial ornithine transcarbamylase deficiency (OTC-D) or those with mild or late-onset disease may be underappreciated or undertreated by patients and first-responding clinicians and go unrecognized for many years until a trigger results in a hyperammonemic crisis.6,7
Triggers of hyperammonemic crises include high-protein intake, pregnancy/labor and delivery, illness, surgical interventions, circumstantial life occurrences (i.e., stress), and some medications such as steroids.3,7
Poor adherence to treatment was reported by 67% of physicians to be a barrier or unmet need in the care of patients with UCD.4
UCDs result in a considerable long-term psychosocial impact (i.e., anxiety, guilt, helplessness, etc.) on patients and their caregivers and may impact the consideration for liver transplantation versus medical treatment.8,9 UCDs also impact patients and their families due to requirements for low-protein meal planning and other disruptions in family routines.8
Absorption of PBA is slower when administered as RAVICTI versus sodium phenylbutyrate, resulting in more sustained levels of active downstream metabolites.10,11
Long-term safety data exist for patients on RAVICTI, and RAVICTI is the only UCD treatment that has been shown to provide ammonia control in both the short term (2 or 4 weeks) and long term (at least 12 months) in clinical studies.10,12
RAVICTI is a nearly odorless and nearly tasteless liquid with no pills or powder to prepare.10,12
RAVICTI is contraindicated in patients with known hypersensitivity to phenylbutyrate.10
aThe safety and efficacy of RAVICTI for the treatment of NAGS deficiency have not been established.10
Review efficacy data for adult
and pediatric patients
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References:
1. Lichter-Konecki U, Diaz GA, Merritt JL II, et al. Ammonia control in children with urea cycle disorders (UCDs); phase 2 comparison of sodium phenylbutyrate and glycerol phenylbutyrate. Mol Genet Metab. 2011;103(4):323-329. doi:10.1016/j.ymgme.2011.04.013 2. Lee B, Diaz GA, Rhead W, et al. Blood ammonia and glutamine as predictors of hyperammonemic crises in patients with urea cycle disorder. Genet Med. 2015;17(7):561-568. doi:10.1038/gim.2014.148. 3. Summar ML, Dobbelaere D, Brusilow S, Lee B. Diagnosis, symptoms, frequency and mortality of 260 patients with urea cycle disorders from a 21-year, multicentre study of acute hyperammonaemic episodes. Acta Paediatr. 2008;97(10):1420-1425. doi:10.1111/j.1651-2227.2008.00952.x 4. Enns GM, Porter MH, Francis-Sedlak M, Burdett A, Vockley J. Perspectives on urea cycle disorder management: Results of a clinician survey. Mol Genet Metab. 2019;128(1-2):102-108. doi:10.1016/j.ymgme.2019.07.009 5. Diaz GA, Schulze A, Longo N, et al. Long-term safety and efficacy of glycerol phenylbutyrate for the management of urea cycle disorder patients. Mol Genet Metab. 2019;127(4):336-345. doi:10.1016/j.ymgme.2019.07.004 6. Ah Mew N, Simpson KL, Gropman AL, Lanpher BC, Chapman KA, Summar ML. Urea Cycle Disorders Overview. In: Adam MP, Ardinger HH, Pagon RA, et al., eds. GeneReviews®. Seattle (WA): University of Washington, Seattle; April 29, 2003. 7. 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 8. Cederbaum JA, LeMons C, Rosen M, Ahrens M, Vonachen S, Cederbaum SD. Psychosocial issues and coping strategies in families affected by urea cycle disorders. J. Pediatr. 2001;138(1 suppl):S72-S80. doi:10.1067/mpd.2001.111839 9. Comparative effectiveness of therapy in rare diseases: liver transplantation vs. conservative management of urea cycle disorders. Accessed April 21, 2022. https://www.rarediseasesnetwork.org/cms/ucdc/Get-Involved/Studies/5117 10. RAVICTI (glycerol phenylbutyrate) Oral Liquid [prescribing information] Horizon. 11. Monteleone JPR, Mokhtarani M, Diaz GA, et al. Population pharmacokinetic modeling and dosing simulations of nitrogen-scavenging compounds: disposition of glycerol phenylbutyrate and sodium phenylbutyrate in adult and pediatric patients with urea cycle disorders. J Clin Pharmacol. 2013;53(7):699-710. doi:10.1002/jcph.92 12. 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
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.