Dosing & Uses
oral liquid
- 500mL/bottle
- Contains 100% w/w of triheptanoin
Long-chain Fatty Acid Oxidation Disorders
Indicated as a source of calories and fatty acids for molecularly confirmed long-chain fatty acid oxidation disorders (LC-FAODs)
Assess metabolic requirements by determining patient’s daily caloric intake (DCI) before calculating dose
Triheptanoin provides 8.3 kcal/mL
Total daily dosage (TDD) (mL) = Patient DCI (kcal) x target % dose of DCI divided by 8.3 kcal/mL
Not currently taking a medium-chain triglyceride (MCT) product
- Initiate at a TDD of ~10% DCI divided into at least 4x/day and increase TDD to up to 35% DCI over a period of 2-3 weeks
Switching from another MCT product
- Discontinue use of MCT products prior to initiation
- Initiate at the last tolerated daily dosage of MCT divided into at least 4x/day
- Increase TDD by ~5% DCI every 2-3 days until the target dosage of up to 35% DCI is achieved
Tolerability
- Consider more frequent smaller doses if patient has difficulty tolerating 25% of the TDD
- Monitor total caloric intake during dosage titration, especially in patients with gastrointestinal (GI) adverse reactions, and adjust all components of the diet as needed
- If patient experiences GI adverse reaction(s), consider dosage reduction until the GI symptoms resolve
- If unable to achieve the target daily dosage of up to 35% DCI during titration, maintain at the maximum tolerated dosage
Dosage Considerations
Patients may require an increase in total fat intake to reach target daily dose
All patients should be under the care of a clinical specialist knowledgeable in appropriate disease-related dietary management based on current nutritional recommendations
oral liquid
- 500mL/bottle
- Contains 100% w/w of triheptanoin
Long-chain Fatty Acid Oxidation Disorders
Indicated as a source of calories and fatty acids for molecularly confirmed long-chain fatty acid oxidation disorders (LC-FAODs)
Assess metabolic requirements by determining patient’s daily caloric intake (DCI) before calculating dose
Triheptanoin provides 8.3 kcal/mL
Total daily dosage (TDD) (mL) = Patient DCI (kcal) x target % dose of DCI divided by 8.3 kcal/mL
Not currently taking a medium-chain triglyceride (MCT) product
- Initiate at a TDD of ~10% DCI divided into at least 4x/day and increase TDD to up to 35% DCI over a period of 2-3 weeks
Switching from another MCT product
- Discontinue use of MCT products prior to initiation
- Initiate at the last tolerated daily dosage of MCT divided into at least 4x/day
- Increase TDD by ~5% DCI every 2-3 days until the target dosage of up to 35% DCI is achieved
Tolerability
- Consider more frequent smaller doses if patient has difficulty tolerating 25% of the TDD
- Monitor total caloric intake during dosage titration, especially in patients with gastrointestinal (GI) adverse reactions, and adjust all components of the diet as needed
- If patient experiences GI adverse reaction(s), consider dosage reduction until the GI symptoms resolve
- If unable to achieve the target daily dosage of up to 35% DCI during titration, maintain at the maximum tolerated dosage
Dosage Considerations
Patients may require an increase in total fat intake to reach target daily dose
All patients should be under the care of a clinical specialist knowledgeable in appropriate disease-related dietary management based on current nutritional recommendations
Interactions
Interaction Checker
No Results
Contraindicated
Serious - Use Alternative
Significant - Monitor Closely
Minor
Contraindicated (0)
Serious - Use Alternative (1)
- orlistat
orlistat decreases effects of triheptanoin by Other (see comment). Avoid or Use Alternate Drug. Comment: Coadministration of triheptanoin with orlistat may reduce exposure to the triheptanoin metabolite, heptanoate, and decrease the effects of triheptanoin.
Monitor Closely (0)
Minor (0)
Adverse Effects
>10%
Abdominal pain (60%)
Diarrhea (44%)
Vomiting (44%)
Nausea (14%)
Warnings
Contraindications
None
Cautions
Feeding tube performance and functionality can degrade over time depending on usage and environmental conditions
Avoid use with pancreatic insufficiency; low or absent pancreatic enzymes may result in reduced heptanoate absorption, and subsequently lead to insufficient medium-chain fatty acid supplementation
Drug interaction overview
- Avoid coadministration with pancreatic lipase inhibitors (eg, orlistat), owing to reduced efficacy and reduced exposure to heptanoate (triheptanoin metabolite)
Pregnancy & Lactation
Pregnancy
No data available on use in pregnant females
Advise females to report pregnancies to Ultragenyx Pharmaceutical Inc at 1-888-756-8657
Lactation
There are no data on the presence of triheptanoin or its metabolites in human or animal milk, effects on breastfed infants, or effects on milk production
MCT and other fatty acids are normal components of breastmilk and the composition of breastmilk varies within feedings, over stages of lactation, and between mothers and populations, owing to maternal factors including genetics, environment, and diet
Pregnancy Categories
A: Generally acceptable. Controlled studies in pregnant women show no evidence of fetal risk.
B: May be acceptable. Either animal studies show no risk but human studies not available or animal studies showed minor risks and human studies done and showed no risk. C: Use with caution if benefits outweigh risks. Animal studies show risk and human studies not available or neither animal nor human studies done. D: Use in LIFE-THREATENING emergencies when no safer drug available. Positive evidence of human fetal risk. X: Do not use in pregnancy. Risks involved outweigh potential benefits. Safer alternatives exist. NA: Information not available.Pharmacology
Mechanism of Action
MCT consisting of three odd-chain 7-carbon length fatty acids (heptanoate)
Provides a source of calories and fatty acids to bypass the LC-FAOD enzyme deficiencies for energy production and replacement
Absorption
Peak plasma concentration
- Single dose: 178.9 micromol/L (0.3 g/kg-dose); 259.1 micromol/L (0.4 g/kg-dose)
- Multiple doses: 319.9 micromol/L (TDD: 1.3 g/kg/day)
Peak plasma time
- Single dose: 0.5 hr (0.3 g/kg-dose); 0.8 hr (0.4 g/kg-dose)
- Multiple doses: 1.2 hr (TDD: 1.3 g/kg/day)
AUC
- Single dose: 336.5 micromol⋅hr/L (0.3 g/kg-dose); 569.1 micromol⋅hr/L (0.4 g/kg-dose)
- Multiple doses: 789.8 micromo⋅hr/L (TDD: 1.3 g/kg/day)
Distribution
Protein bound: ~80% (heptanoate)
Metabolism
Heptanoate, formed by hydrolysis of triheptanoin, can be metabolized to beta-hydroxypentanoate (BHP) and beta-hydroxybutyrate (BHB) in the liver
Elimination
Half-life: Not determined owing to multiple peak concentrations observed
Excretion: Minimally excreted in urine (triheptanoin and metabolites)
Clearance
0.3 g/kg-dose: 6.05 L/hr/kg
0.4 g/kg-dose: 4.31 L/hr/kg
Administration
Compatibility
Oral compatibility
- Use containers, dosing syringes, or measuring cups made of compatible materials suchas stainless steel, glass, high-density polyethylene (HDPE), polypropylene, low-densitypolyethylene, polyurethane, and silicone
- Not compatible with certain plastics; do not use containers, dosing syringes, or measuring cups made of polystyrene or polyvinyl chloride (PVC) plastics
- Monitor containers, dosing components, or utensils that are in contact with triheptanoin to ensure proper functioning and integrity
Feeding tube compatibility
- Administer via oral or enteral feeding tubes manufactured of silicone or polyurethane
- Do not use PVC feeding tubes
- Feeding device performance and functionality can degrade over time depending on usage and environmental conditions
- Monitor feeding tube to ensure proper functioning and integrity
Oral Preparation
Use an oral syringe or measuring cup made of compatible materials as listed above to withdraw prescribed volume from the bottle
Mix into semisolid foods and liquids (eg, plain or artificially sweetened fat-free yogurt, fat-free milk, formula, cottage cheese, whole grain hot cereal, fat-free low-carbohydrate pudding, smoothies, applesauce, baby food)
Add prescribed amount to a clean bowl, cup, or container, which contains an appropriate amount of semisolid food or liquid that takes into consideration the age, size, and average consumption of the patient in order to ensure administration of the full dose
Mix thoroughly into the food or liquid
Oral Administration
Administer orally or enterally via a silicone or polyurethane feeding tube
Administer with food to avoid GI upset
Feeding tube
- Draw up entire amount of formula mixture into a slip-tip syringe
- Remove residual air from the syringe and connect syringe directly into the feeding tube feeding port
- Push contents into the feeding tube feeding port using steady pressure until empty
- Flush feeding tubes with 5-30 mL of water
- Modify flush volume based on specific patient needs and in cases of fluid restriction
- Discard any unused portion; do not save for later use
- Patients receiving bolus delivery of enteral feeds: Administer over 15-20 minutes
- Patients receiving continuous feeds: Administer over 30-60 minutes alternating with formula alone
Missed dose
- Take the next dose as soon as possible with subsequent doses taken at 3- to 4hr intervals
- Skip missed dose if unable to take all 4 doses in a day
Storage
Unopened bottle
- Store at 20-25ºC (68-77ºF); excursions permitted to 15-30ºC (59-86ºF)
- Dispense only in glass or HDPE bottles
- Do not freeze
Opened bottles
- Discard after 90 days of opening, but not beyond the expiration date on the bottle
- Do not dose or store in polystyrene or PVC containers
Prepared doses mixed with food or liquid
- Refrigerate for up to 24 hr
Images
Formulary
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