triheptanoin
Orphan DrugFDA Approved, EMA Approved
Description
Triheptanoin is a medium odd-chain triglyceride that provides an alternative energy source for patients with long-chain fatty acid oxidation disorders. It has been investigated for use in glucose transport disorders as it can provide anaplerotic substrates that bypass glucose metabolism defects. The drug is approved for treatment of molecularly confirmed long-chain fatty acid oxidation disorders.
Indications & Therapeutic Use
glucose transport disorder, long-chain fatty acid oxidation disorders
Global Availability (6 countries)
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Available Through
This drug is available for procurement through the following fulfilment partner.
Fulfilment Partner
triheptanoin
| Generic Name | triheptanoin |
| Brands | 1 brand available |
| Active Ingredient | triheptanoin |
| Drug Class | glucose transport disorder |
| Manufacturer | Ultragenyx Pharmaceutical |
| Dosage Forms | oral liquid, 1000mg/mL |
| Medical Code | A16AX12 |
| Orphan Status | Yes — Orphan Drug |
| Cold Chain | Not Required |
| Lead Time | 21 days |
| Reg. Status | FDA Approved, EMA Approved |
| Clinical Trial | NCT02214160 |
| Countries | 6 countries |
Authority Signals
Last Verified3/21/2026
Data ConfidenceHigh (AI + Pharmacist)
Source Citations7 Validated Nodes