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Glycerol Phenylbutyrate (Ravicti) [Internet]. Ottawa (ON): Canadian Agency for Drugs and Technologies in Health; 2017 Apr.
Post-hoc evaluations of blood ammonia levels were performed in patients who were diagnosed with urea cycle disorders (UCDs) during infancy (early onset) and after infancy (late onset). For patients with UCD onset at two years of age or younger, the mean blood ammonia AUC0–24 levels were 17% lower with glycerol phenylbutyrate (GPB) treatment versus sodium phenylbutyrate (NaPBA) treatment. For patients with UCD onset after two years of age, the mean blood ammonia AUC0–24 levels were lower by 10% with GPB versus NaPBA.
| Onset ≤ 2 years | Onset > 2 years | |||
|---|---|---|---|---|
| GPB (n = 10) | NaPBA (n = 10) | GPB (n = 34) | NaPBA (n = 34) | |
| AUC0–24 of Blood Ammonia Levels, μmol h/L | ||||
| Mean (SD) | 732.84 (604.13) | 878.91 (586.41) | 904.97 (679.73) | 1005.37 (937.14) |
| Between-group difference | −146.07 (205.85) | −100.40 (651.70) | ||
| P value for between-group comparison | 0.052 | 0.376 | ||
| Cmax of Blood Ammonia Levels, μmol/L | ||||
| Mean (SD) | 51.62 (36.92) | 60.23 (45.27) | 63.68 (48.76) | 73.94 (72.08) |
| Between-group difference | −8.61 (23.11) | −10.27 (47.68) | ||
| P value for between-group comparison | 0.269 | 0.218 | ||
AUC = area under the curve; GPB = glycerol phenylbutyrate; NaPBA = sodium phenylbutyrate; SD = standard deviation; UCD = urea cycle disorder.
Source: Clinical Study Report of HPN-100-006.24
A post-hoc analysis of glutamine levels was conducted in HPN-100-006. Mean ± standard deviation (SD) glutamine values were statistically significantly lower (P = 0.031, paired t-test; P = 0.017, Wilcoxon signed rank test) after GPB treatment compared with NaPBA treatment (757.7 ± 237.44 μmol/L versus 808.9 ± 251.48 μmol/L, respectively) in the safety population.
| GPB | NaPBA | |
|---|---|---|
| Safety population | N = 44 | N = 44 |
| Mean (SD) | 757.7 (237.44) | 808.9 (251.48) |
| Change from baseline to study end point | −57.1 (202.88) | 41.7 (163.11) |
| Difference between GPB and NaPBA, mean (SD) | −51.2 (152.23) | |
| P value | 0.031 | |
GPB = glycerol phenylbutyrate; NaPBA = sodium phenylbutyrate; SD = standard deviation.
Source: Clinical Study Report of HPN-100-006.24
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