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Background  Hyperammonaemia is a key sign of decompensation in organic acidurias (OAs) and can contribute to severe neurological complications, thus requiring rapid treatment.    Methods  A  post-hoc  analysis of two retrospective studies analysed the efficacy of carglumic acid ± ammonia (NH 3 ) scavengers compared with scavengers alone for reducing plasma NH 3  levels in patients with OAs and hyperammonaemia (plasma NH 3  > 60 μmol/L) during decompensation episodes. NH 3  was analysed in 12-h periods at 0–48 h and 24-h periods at 48–120 h. Treatment-emergent adverse events (TEAEs) were recorded.    Results  Of 98 episodes, 38 were treated with carglumic acid (34 patients), 33 with NH 3  scavengers (22 patients) and 27 with carglumic acid combined with NH 3  scavengers (27 patients). Overall, 45% (carglumic acid group), 46% (NH 3  scavengers group) and 74% (combination group) of episodes occurred in neonates. Median episode duration was 6 days for the carglumic acid and combination groups, and 9 days for the NH 3  scavenger group. Median baseline NH 3  level was: 199 μmol/L, carglumic acid; 122 μmol/L, NH 3  scavengers; and 271 μmol/L, combination; 13, 30 and 11% of episodes required extracorporeal detoxification (ED), respectively. Data were censored at ED initiation. While baseline NH 3  levels were higher in the combination and carglumic acid groups, mean reduction in NH 3  levels to 72 h in both groups was greater than the NH 3  scavengers’ group; reductions were greatest in the combination group.  Mean change in plasma NH 3  vs baseline in the carglumic acid, NH 3  scavengers and combination groups, respectively, was − 13, + 12% and − 27% at 0–12 h ( p  < 0.05 NH 3  scavengers vs combination); − 47, − 22% and − 52% at 12–24 h (not significant); − 44, − 5% and − 61% at 24–48 h; and − 66, − 16% and − 76% at 48–72 h ( p  < 0.05 carglumic acid/combination groups vs NH 3  scavengers for both timepoints). The number of TEAEs was similar between groups and mainly related to the disease/condition.    Conclusions  Carglumic acid is a well-tolerated and efficacious treatment for OA decompensation episodes. When given alone or combined with NH 3  scavengers, the reduction in NH 3  was greater than with NH 3  scavengers alone in the first 72 h.

Effect of carglumic acid with or without ammonia scavengers on hyperammonaemia in acute decompensation episodes of organic acidurias