Triheptanoin
Regulatory sources consulted
Approved indications
- Source of calories and fatty acids for adults and paediatric patients with molecularly confirmed long-chain fatty-acid oxidation disorders.
Clinical warnings
- Major warning · Do not administer in pancreatic insufficiency: low or absent pancreatic enzymes may reduce absorption and clinical effect. — https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=5644d49f-08b9-e806-e063-6294a90a80f9
- Major warning · Monitor feeding-tube function and integrity; do not use PVC tubes or components and do not administer the liquid alone. — https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=5644d49f-08b9-e806-e063-6294a90a80f9
Drug interactions
- HighPancreatic lipase inhibitors, such as orlistatA08AB01
Mechanism: They may reduce exposure to the heptanoate metabolite and the clinical effect of triheptanoin.
Recommendation: Avoid concomitant administration.
https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=5644d49f-08b9-e806-e063-6294a90a80f9
Adverse events
Common (≥1%)
Abdominal pain · Diarrhoea · Vomiting · Nausea
Pregnancy and lactation
FDA category: Datos humanos insuficientes
There are insufficient data to assess risk during pregnancy; a pregnancy exposure registry is available. Transfer into human milk is unknown; weigh the benefits of breastfeeding against the mother's clinical need.
Recent literature (PubMed)
Paroxysmal movement disorders are common in Glut1 deficiency syndrome (Glut1DS). Not all patients respond to or tolerate ketogenic diets. The objective was to evaluate the effectiveness and safety of triheptanoin in reducing the frequency of disabling movement disorders in patients with Glut1DS not receiving a ketogenic diet. UX007G-CL301 was a randomized, double-blind, placebo-controlled, phase 3 crossover study. After a 6-week run-in, eligible patients were randomized 1:1 to the first sequence (triheptanoin/placebo or placebo/triheptanoin) titration plus maintenance, followed by washout and the opposite sequence titration plus maintenance. The placebo (safflower oil) matched the appearance, taste, and smell of triheptanoin. Open-label triheptanoin was administered in the extension. The frequency of disabling paroxysmal movement disorder events per 4 weeks (recorded by diary during maintenance; primary endpoint) was assessed by Wilcoxon rank-sum test. Forty-three patients (children, n = 16; adults, n = 27) were randomized and treated. There was no difference between triheptanoin and placebo in the mean (interquartile range) number of disabling paroxysmal movement disorder events (14.3 [4.7-38.3] vs. 11.8; [3.2-28.7]; Hodges-Lehmann estimated median difference: 1.46; 95% confidence interval, -1.12 to 4.36; P = 0.2684). Treatment-emergent adverse events were mild/moderate in severity and included diarrhea, vomiting, upper abdominal pain, headache, and nausea. Two patients discontinued the study because of non-serious adverse events that were predominantly gastrointestinal. The study was closed early during the open-label extension because of lack of effectiveness. Seven patients continued to receive triheptanoin compassionately. There were no significant differences between the triheptanoin and placebo groups in the frequency of disabling movement disorder events during the double-blind maintenance period. © 2024 The Authors. Movement Disorders published by Wiley Per