Developing a new formulation of sodium phenylbutyrate

Archives of Disease in Childhood

Volumn 97, Issue 12, 2012, Pages 1081-1085

  Guffon, Nathalie ^a   Kibleur, Yves ^b   Copalu, William ^c   Tissen, C ^d   Breitkreutz, Joerg ^d  

^a HOSPICES CIVILS DE LYON   (France)

^b Lucane Pharma   (France)

^c PAREXEL INTERNATIONAL   (United States)

^d HEINRICH HEINE UNIVERSITY   (Germany)

Author keywords

[No Author keywords available]

Indexed keywords

4 PHENYLBUTYRIC ACID;

ADULT; AGEUSIA; AREA UNDER THE CURVE; ARTICLE; BIOEQUIVALENCE; BITTER TASTE; CONTROLLED STUDY; DRUG FORMULATION; DRUG INDUCED HEADACHE; DRUG RELEASE; DRUG SAFETY; DRUG SOLUBILITY; DRUG TOLERABILITY; FEMALE; HUMAN; HUMAN EXPERIMENT; IN VITRO STUDY; MALE; MAXIMUM PLASMA CONCENTRATION; MOLECULE; NORMAL HUMAN; PRIORITY JOURNAL; TASTE; TASTE BUD; VOMITING; WEAKNESS;

ADULT; BIOLOGICAL AVAILABILITY; CHEMISTRY, PHARMACEUTICAL; FEMALE; HUMANS; MALE; MIDDLE AGED; PHENYLBUTYRATES; TASTE; THERAPEUTIC EQUIVALENCY; YOUNG ADULT;

EID: 84869861876     PISSN: 00039888     EISSN: 14682044     Source Type: Journal    
DOI: 10.1136/archdischild-2012-302398     Document Type: Article

References (25)

1. Brusilow SW, Maestri NE. Urea cycle disorders: diagnosis, pathophysiology, and therapy. AdvPediatr 1996;43:127-70.
2. Lewis HB. Studies in the synthesis of hippuric acid after benzoate ingestion in man. J BiolChem 1914;18:225.
3. Sherwin CP, Kennard KS. Toxicity of phenylacetic acid. J Biol Chem 1919;17:259-64.
4. Brusilow W, Tinker J, Batshaw ML. Amino acid acylation: a mechanism of nitrogen excretion in inborn errors of urea synthesis. Science 1980;207:659-64.
5. Summary of Product Characteristics, AMMONAPS 940 mg/g granules, Swedish Orphan International AB, Sweden.
6. El-Chaar GM, Mardy G, Wehlou K, et al. Randomized, double blind comparison of brand and generic antibiotic suspensions: II. A study of taste and compliance in children. Pediatr Infect Dis J 1996;15:18-22.
7. European Paediatric Formulation Initiative (EuPFI) Conference Formulating Better Medicines for Children http://www.eupfi.org(EuPFI, Berlin, September 2010).
8. Nunn T, Williams J. Formulation of medicines for children. Br J Clin Pharmacol 2005;59:674-6.
9. Royal College of Paediatrics and Child Health, Safer and Better Medicines for Children. 2004.
10. Menella J, Beauchamp G. Optimizing oral medications for children. Clin Ther 2008;30:2120-32.
11. Maestri NE. Prospective treatment of urea cycle disorders. J Pediatr 1991;6:923-26.
12. Dover GJ, Brusilow S, Charache S. Induction of fetal hemoglobin production in subjects with sickle cell anemia by oral sodium phenylbutyrate. Blood 1994;84:339-43.
13. Lee B, Rhead W, Diaz GA, et al. Phase 2 comparison of a novel ammonia scavenging agent with sodium phenylbutyrate in patients with urea cycle disorders: safety, pharmacokinetics and ammonia control. Mol Genet Metab 2010;100:221-8.
14. Maestri NE, Brusilow SW, Clissold DB, et al. Long-term treatment of girls with ornithine transcarbamylase deficiency. N Engl J Med 1996;335:855-9.
15. Maestri NE, Clissold DB, Brusilow SW. Long-term survival of patients with argininosuccinate synthetase deficiency. J Pediatr 1995;127:929-35.
16. Msall M, Batshaw ML, Suss R, et al. Neurologic outcome in children with inborn errors of urea synthesis. Outcome of urea-cycle enzymopathies. N Engl J Med 1984;310:1500-5.
17. Gilbert J, Baker SD, Bowling MK, et al. A phase I dose escalation and bioavailability study of oral sodium phenylbutyrate in patients with refractory solid tumor malignancies. Clin Cancer Res 2001;7:2292-300.
18. Collins AF, Pearson HA, Giardina P, et al. Oral sodium phenylbutyrate therapy in homozygous beta thalassemia: a clinical trial. Blood 1995;85:43-9.
19. Feillet F, Leonard JV. Alternative pathway therapy for urea cycle disorders. J Inherit Metab Dis 1998;21(Suppl 1):101-11.
20. Caruthers RL, Johnson CE. Stability of extemporaneously prepared sodium phenylbutyrate oral suspensions. Am J Health Syst Pharm. 2007;64:1513-15.
21. Woertz K, Tissen C, Kleinebudde P, et al. Taste sensing systems (electronic tongues) for pharmaceutical applications. Int J Pharm 2011;417:256-71.
22. Garsuch V, Breitkreutz J. Novel analytical methods for the characterization of oral wafers. Eur J Pharm Biopharm 2009;73:195-201.
23. Woertz K, C. Tissen C, Kleinebudde P, et al. Performance qualification of an electronic tongue according to ICH guideline Q2. J Pharm Biomed Anal 2010;51:497-506.
24. http://www.ema.europa.eu/docs/en-GB/document-library/EPAR-Scientific- Discussion/human/000219/WC500024748.pdf
25. Guideline on the investigation of bioequivalence (CPMP/QWP/EWP/1401/98 Rev. 1). European Medicines Agency (EMA), 2010:1-27. http://www.emea.europa.eu/ docs/en-GB/document-library/Scientific-guideline/2010/01/WC500070039.pdf.