Summary

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• This summary is based on the review of five systematic review(s)/meta-analysis(es). ^[1-5]
• In patients with long-chain fatty acid oxidation disorders (LC-FAODs), triheptanoin reduced major clinical events (MCEs) by 86% in the triheptanoin-naive cohort, decreasing the median rate from 2.00 to 0.28 events/patient/year (p = 0.0343). In the rollover cohort, there was a 43% reduction in MCEs, from 1.76 to 1.00 events/patient/year (p = 0.0347).
• In a long-term extension study, triheptanoin reduced the mean annualized MCE rate from 1.76 to 0.96 events/year (p = 0.0319) in the rollover group, with a 66% reduction in median MCE duration. The triheptanoin-naive group experienced an 80% reduction in MCE duration.
• In phosphofructokinase deficiency (PFKD), triheptanoin did not significantly improve exercise performance or heart rate, despite an increase in palmitate production and utilization during exercise.
• In the NCT02214160 (CL202) study, 68.1% of patients experienced treatment-emergent adverse events (TEAEs), mostly mild to moderate. Serious TEAEs, including diverticulitis, ileus, and rhabdomyolysis, were reported in five patients, but all resolved.
• In the long-term extension study, common adverse events included diarrhea, abdominal pain, and vomiting. Two patients had adverse events leading to death, but neither was drug-related.