Is Triheptanoin approved by the FDA?

Yes, triheptanoin, marketed under the brand name Dojolvi, is FDA approved. The U.S. Food and Drug Administration (FDA) approved Dojolvi on June 30, 2020, for the treatment of long-chain fatty acid oxidation disorders (LC-FAODs) in adults and children.

What is Triheptanoin?

Triheptanoin is an oral liquid medication used to treat long-chain fatty acid oxidation disorders. These are rare genetic disorders that affect the body's ability to break down certain fats and convert them into energy, potentially leading to symptoms such as low blood sugar, muscle weakness, heart problems, and other life-threatening issues.

How is Triheptanoin Administered?

Triheptanoin is typically taken at least four times per day with meals or snacks. It must be mixed well with liquid or soft food to prevent stomach upset. Examples of suitable mixers include:

• Fat-free yogurt (plain or flavored)
• Fat-free pudding or smoothies
• Low-sugar applesauce
• Fat-free cottage cheese
• Skim (fat-free) milk
• Fat-free baby food
• Whole grain hot cereal

Key Warnings and Precautions

Pancreatic Problems:

• Patients with pancreatic issues should inform their doctor as triheptanoin may not work as effectively if digestion is compromised.

Pregnancy and Breastfeeding:

• It is important to inform your doctor if you are pregnant or breastfeeding. Pregnant women may be enrolled in a pregnancy safety study to track the effects of triheptanoin on the baby.

Side Effects

Common Side Effects:

• Nausea
• Vomiting
• Stomach pain
• Diarrhea

Serious Allergic Reactions:

• Hives
• Difficulty breathing
• Swelling of the face, lips, tongue, or throat

Diet and Storage:

• Patients may need to follow a special diet while using triheptanoin. The medicine should be stored in its original container at room temperature, away from moisture and heat, and should not be frozen. Mixed medicine should be refrigerated and used within 24 hours. Any unused triheptanoin should be discarded 90 days after opening or on the expiration date, whichever comes first.

Conclusion

Triheptanoin (Dojolvi) is an FDA-approved treatment for long-chain fatty acid oxidation disorders, approved on June 30, 2020. It is a vital medication for managing these rare genetic disorders, ensuring that the body can effectively convert fats into energy, thereby preventing severe health issues. Proper administration and adherence to dietary recommendations are crucial for its effectiveness and patient safety. Always consult with a healthcare provider for personalized medical advice and follow all prescribed guidelines.

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