Profile of sodium phenylbutyrate granules for the treatment of urea-cycle disorders: patient perspectives

Figures & data

Figure 1 Alternative route of ammonia removal by phenylacetate/phenylbutyrate action on glutamine.

Table 1 Summary of available treatments for urea-cycle disorders

Active principal	Brand/manufacturer	Administration/dosage	Disadvantages	Advantages	References
Sodium benzoate	Pharma international	Oral, 500 mg tablet	Poor adherence		^Citation7
Sodium benzoate	Pharma international	Oral, 100 mg/mL	Poor adherence	Adapted for children	^Citation7
Sodium benzoate	Pharma international	IV, 2 g/10 mL		Independent dosing	^Citation7
Sodium benzoate and sodium PB	Ammonul (Valeant Pharmaceuticals)	IV, 10% and 10%			^Citation26
Sodium PB	Pharma international	IV, 2 g/10 mL		Independent dosing	^Citation7
Sodium PB	Ammonaps (Swedish Orphan Biovitrum)	Oral, 500 mg tablet	Poor adherence	Adapted for children	^Citation39
Sodium PB	Buphenyl (Horizon Pharma)	Oral, 250 mg tablet	Poor adherence	Adapted for children	^Citation7
Sodium PB	Buphenyl (Horizon Pharma)	Powder, 3.2 g/3 g	Poor adherence	Adapted for children	^Citation7
Sodium PB	Pheburane (Lucane Pharma)	Granules	No flavor	Needs collaboration by patient	^Citation29, ^Citation44
Glycerol PB	Ravicti (Horizon Pharma)	1 mg/mL	No flavor	Adapted for all ages	^Citation48

Abbreviations: IV, intravenous; PB, phenylbutyrate.

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