triheptanoin

Triheptanoin is a type of fat (triglyceride) used in the treatment of pediatric and adult patients with molecularly confirmed long-chain fatty acid oxidation disorders (LC-FAOD). LC-FAOD are rare, genetic life-threatening disorders that disrupt the body’s ability to generate energy from long-chain fatty acids, leading to chronic deficiency of energy. Triheptanoin is a synthetic medium chain triglyceride that is used to compensate for the fatty acid deficiency caused by LC-FAOD and acts as a source of calories and fatty acids.

LC-FAOD is caused by mutations in the genes that encode mitochondrial enzymes required for breaking up long-chain fatty acids to generate energy. The gene mutations lead to absence or deficiency of these enzymes and inability to metabolize long chain fatty acids. Triheptanoin is a medium-chain triglyceride that can be directly metabolized for energy generation by the cells, bypassing the process of long-chain fatty acid oxidation and the requirement for enzymes that enable the process. Triheptanoin helps reduce the complications associated with LC-FAOD.

Triheptanoin is an oral solution that is mixed with liquids or semi-solid foods and taken orally. In patients receiving nutrition through a feeding tube triheptanoin is directly injected into the feeding tube feeding port.

• All patients receiving triheptanoin should be under the care of a clinical specialist knowledgeable in appropriate disease-related dietary management based on current nutritional recommendations.
• Feeding tube functionality and performance can deteriorate over time and contribution from triheptanoin cannot be ruled out.
  • Do not administer triheptanoin in polyvinyl chloride feeding tubes.
  • Flush the feeding tubes with water after administering triheptanoin. Flush volume should be based on the individual patient’s needs.
  • Regularly monitor the integrity and proper functioning of the feeding tube.
• Do not use triheptanoin to treat patients with pancreatic insufficiency. Pancreatic enzymes break down triheptanoin and release heptanoate as medium-chain fatty acids in the intestines. Low levels or absence of pancreatic enzymes can reduce heptanoate absorption, and consequently, may not adequately meet the patient’s requirement of medium-chain fatty acids.

Common side effects of triheptanoin include:

• Abdominal discomfort
• Abdominal pain
• Abdominal distension
• Gastrointestinal pain
• Diarrhea
• Vomiting
• Nausea

Call your doctor immediately if you experience any of the following symptoms or serious side effects while using this drug:

• Serious heart symptoms include fast or pounding heartbeats, fluttering in your chest, shortness of breath, and sudden dizziness;
• Severe headache, confusion, slurred speech, severe weakness, vomiting, loss of coordination, feeling unsteady;
• Severe nervous system reaction with very stiff muscles, high fever, sweating, confusion, fast or uneven heartbeats, tremors, and feeling like you might pass out; or
• Serious eye symptoms include blurred vision, tunnel vision, eye pain or swelling, or seeing halos around lights.

This is not a complete list of all side effects or adverse reactions that may occur from the use of this drug. Call your doctor for medical advice about serious side effects or adverse reactions. You may also report side effects or health problems to the FDA at 1-800-FDA-1088.

Oral liquid

• 500 mL/bottle
• Contains 100% w/w of triheptanoin

Adult and Pediatric:

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 4 times/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 4 times/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

Overdose

There is no information available on triheptanoin overdose.

Inform your doctor of all medications you are currently taking, who can advise you on any possible drug interactions. Never begin taking, suddenly discontinue, or change the dosage of any medication without your doctor’s recommendation.

• Triheptanoin has no listed severe interactions with other drugs.
• Serious interactions of triheptanoin include:
  • orlistat
• Triheptanoin has no listed moderate interactions with other drugs.
• Triheptanoin has no listed mild interactions with other drugs.

The drug interactions listed above are not all of the possible interactions or adverse effects. For more information on drug interactions, visit the RxList Drug Interaction Checker.

It is important to always tell your doctor, pharmacist, or health care provider of all prescription and over-the-counter medications you use, as well as the dosage for each, and keep a list of the information. Check with your doctor or health care provider if you have any questions about the medication.

• There are no data available on the use of triheptanoin in pregnant women to identify a drug-associated risk of major birth defects or miscarriage.
• There is no information on the presence of triheptanoin or its metabolites in animal milk or breastmilk, or its effects on milk production or the breastfed infant.
• Medium-chain triglycerides and other fatty acids are normal components of breastmilk and the composition varies based on various factors.
• Decision to breastfeed should be based on the nursing mother’s clinical need for triheptanoin, health and developmental benefits of breastfeeding, and the risks to the breastfed infant from exposure to the drug or the mother’s underlying condition.
• There is a pregnancy safety study that collects pregnancy outcome data in women taking triheptanoin during pregnancy. Pregnant patients can enroll in the study by calling 1-888-756-8657.

• Prepare and take triheptanoin exactly as per instructions.
• Inspect the feeding tube regularly and report to your healthcare provider if the feeding tube is degraded or fails to function properly.
• Keep triheptanoin safely out of reach of children.
• In case of overdose, report your symptoms to your healthcare provider or to the Poison Control.