Triheptanoin

Generic Name
Triheptanoin
Brand Names
Dojolvi
Drug Type
Small Molecule
Chemical Formula
C24H44O6
CAS Number
620-67-7
Unique Ingredient Identifier
2P6O7CFW5K
Background

Triheptanoin is a source of heptanoate fatty acids, which can be metabolized without the enzymes of long chain fatty acid oxidation. In clinical trials, patients with long chain fatty acid oxidation disorders (lc-FAODs) treated with triheptanoin are less likely to develop hypoglycemia, cardiomyopathy, rhabdomyolysis, and hepatomegaly. Complications in lc-FAOD patients are reduced from approximately 60% to approximately 10% with the addition of triheptanoin.

Triheptanoin was granted FDA approval on 30 June 2020.

Indication

Triheptanoin is a medium chain triglyceride indicated to provide calories and fatty acids to treat long chain fatty acid oxidation disorders (lc-FAODs).

Associated Conditions
Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)
Associated Therapies
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Ultragenyx Reports Third Quarter 2024 Financial Results and Corporate Update

Ultragenyx reported Q3 2024 revenue of $139 million, up 42% YoY, with Crysvita® at $98 million and Dojolvi® at $21 million. The company reaffirmed 2024 revenue guidance of $530-$550 million and announced Breakthrough Designation for setrusumab (UX143) in osteogenesis imperfecta. DTX401 Phase 3 data showed a 62% mean reduction in cornstarch in crossover patients with glycogen storage disease type Ia (GSDIa).
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