Acts as a medium-chain triglyceride consisting of three odd-chain 7-carbon length fatty acids (heptanoate) that provide a source of calories and fatty acids to bypass the long-chain fatty acid oxidation disorder enzyme deficiencies for energy production and replacement.
Therapeutic effects:
Reduction in major clinical events associated with long-chain fatty acid oxidation disorders.
Pharmacokinetics⬆⬇
Absorption: Extent of absorption following oral administration unknown.
Distribution: Unknown.
Metabolism/Excretion: Hydrolyzed to heptanoate and glycerol via pancreatic lipases in intestines; heptanoate is metabolized in the liver. Minimally excreted in the urine.
Half-Life: Unknown.
Time/Action Profile⬆⬇
(plasma concentrations)
ROUTE
ONSET
PEAK
DURATION
PO
unknown
0.5–1.2 hr
unknown
Contraind./Precautions⬆⬇
Contraindicated in:
Patients with feeding tubes made of polyvinyl chloride (↑ risk of feeding tube dysfunction);
Pancreatic insufficiency.
Use Cautiously in:
OB: Safety not established in pregnancy;
Lactation: Safety not established in breastfeeding.
Total daily dose of triheptanoin (mL) = (Patient's daily caloric intake [kcal] × desired % of daily caloric intake) divided by 8.3 kcal/mL.
PO (Adults ): Not currently receiving a medium-chain triglyceride product: ~10% of the patient's total prescribed daily caloric intake divided into at least 4 times daily; ↑ dose by ~5% of the patient's total prescribed daily caloric intake every 2–3 days until target dose of up to 35% of the patient's total prescribed daily caloric intake is achieved. Switching from another medium-chain triglyceride product: Initiate at the last tolerated daily dosage of medium-chain triglyceride divided into at least 4 times daily; ↑ dose by ~5% of the patient's total prescribed daily caloric intake every 2–3 days until target dose of up to 35% of the patient's total prescribed daily caloric intake is achieved.
Availability⬆⬇
Oral solution: 100% (w/w)
Assessment⬆⬇
Assess feeding tube site and function prior to administration; monitor regularly to ensure proper functioning and integrity.
Monitor for signs and symptoms of GI adverse reactions (nausea, vomiting, diarrhea, abdominal pain, abdominal distention). Consider dosage ↓ until the GI symptoms resolve. If a patient is unable to achieve the target daily dosage of up to 35% daily caloric intake during dosage titration, maintain the patient at the maximum tolerated dosage. Correct any significant fluid and/or electrolyte abnormalities in patients with severe diarrhea and/or vomiting. Monitor serum electrolytes in patients with significant vomiting and/or diarrhea.
Implementation⬆⬇
Determine daily caloric intake before calculating the dose of triheptanoin. Round the total daily dosage to the nearest mL; divide the total daily dosage into at least four approximately equal individual doses. Patients may require an ↑ in their total fat intake to reach a target daily dosage.
Discontinue all other medium-chain triglyceride products prior to the 1st dose of triheptanoin.
Do not administer therapy through feeding tubes manufactured of polyvinyl chloride. Prepare or administer dose using containers, oral syringes, or measuring cups made of compatible materials such as stainless steel, glass, high-density polyethylene, polypropylene, low-density polyethylene, polyurethane, or silicone.
PO: Mix thoroughly with semisolid food or liquids such as plain or artificially sweetened fat-free yogurt; fat-free milk, formula, or cottage cheese; whole grain hot cereal; fat-free low-carbohydrate pudding; smoothies; applesauce; or baby food. May be thoroughly mixed with tube feeding formula or medical food prior to administering via feeding tube, Y-connector, or feeding tube extension set made of silicone or polyurethane. The amount should be based on the age, size, and fluid needs of the patient in order to ensure administration of the full dose; if a patient has difficulty tolerating ¼ of the total daily dosage at one time, more frequent smaller doses may be considered. Do not administer alone to avoid GI upset and feeding tube degradation. May store unused mixed triheptanoin for up to 24 hr in the refrigerator. If not used within 24 hr, discard the mixture in the trash; do not pour down the sink or save for later use.
If a dose is missed, take the next dose as soon as possible with subsequent doses taken at 3- to 4-hr intervals. Skip the missed dose if it will not be possible to take all doses in a day.
Patient/Family Teaching⬆⬇
Explain purpose and side effects of triheptanoin. Teach patient or caregiver to mix thoroughly into semisolid foods, liquids, or formula. Advise patient or caregiver to avoid using containers or utensils made of polystyrene or polyvinyl chloride plastics when preparing or administering drug. Counsel patient that if a dose is missed, take next dose as soon as possible, with subsequent doses taken at 3- to 4-hr intervals; if this is not possible, take all four doses in a day, skipping the missed dose. Advise patient or caregiver to read Patient Information before starting therapy.
Recommend patient or caregiver regularly inspect the feeding tube for proper functioning and report to the health care provider if any issues are identified.
Advise patient to report and seek treatment for severe abdominal pain, diarrhea, vomiting, and nausea.
Advise patient to notify health care professional of all Rx or OTC medications, vitamins, or herbal products being taken and to consult health care professional before taking other medications.
Rep: Advise women of reproductive potential to notify health care professional if pregnancy is planned or suspected or if breastfeeding. Health care providers are encouraged to register patients in the pregnancy safety study for triheptanoin by calling Ultragenyx Pharmaceuticals at 1-888-756-8657.
Evaluation/Desired Outcomes⬆⬇
Reduction in major clinical events associated with long-chain fatty acid oxidation disorders.
