Drug updated on 5/17/2024
Dosage Form | Liquid (oral; 100% w/w of triheptanoin) |
Drug Class | Medium-chain triglycerides |
Ongoing and Completed Studies | ClinicalTrials.gov |
Indication
- Indicated as a source of calories and fatty acids for the treatment of adult and pediatric patients with molecularly confirmed long-chain fatty acid oxidation disorders (LC-FAOD).
Latest News
![loading GIF](/img/news-loading.gif)
Summary
- Triheptanoin (Dojolvi) is indicated as a source of calories and fatty acids for the treatment of adult and pediatric patients with molecularly confirmed long-chain fatty acid oxidation disorders (LC-FAOD).
- Five randomized controlled studies focused on its efficacy in treating LC-FAODs.
- In these studies, triheptanoin demonstrated significant efficacy by reducing major clinical events such as rhabdomyolysis, hypoglycemia, and cardiomyopathy across different patient cohorts.
- The safety profile of triheptanoin was consistent with previous observations; most adverse events were mild to moderate in severity.
- It did not show significant improvement in heart rate during exercise or exercise performance for patients with phosphofructokinase deficiency nor significantly reduce seizure frequency for those with glucose transporter 1 deficiency syndrome.
- The studies highlight that Dojolvi's effectiveness might vary among different patient subtypes within a disorder spectrum.
Product Monograph / Prescribing Information
Document Title | Year | Source |
---|---|---|
Dojolvi (triheptanoin) Prescribing Information. | 2023 | Ultragenyx Pharmaceutical Inc., Novato, CA |
Randomized Controlled Trials
Sex Distribution:
![No Data](/img/Nodata_Icon.png)
Year:
2023
Source:Journal of Inherited Metabolic Disease
Sex Distribution:
![No Data](/img/Nodata_Icon.png)
Year:
2022
Source:Neuromuscular Disorders
Document Title
Sex Distribution:
![No Data](/img/Nodata_Icon.png)
Year:
2022
Source:Epilepsia
Sex Distribution:
![No Data](/img/Nodata_Icon.png)
Year:
2021
Source:Journal of Inherited Metabolic Disease
Sex Distribution:
![No Data](/img/Nodata_Icon.png)
Year:
2021
Source:Clinical Nutrition ESPEN
Clinical Practice Guidelines
Document Title | Year | Source |
---|---|---|
Diagnosis and management of glycogen storage disease type IV, including adult polyglucosan body disease: a clinical practice resource. | 2023 | Molecular Genetics and Metabolism |
Nutrition management guideline for very-long chain acyl-CoA dehydrogenase deficiency (VLCAD): an evidence- and consensus-based approach. | 2020 | Molecular Genetics and Metabolism |