Drugs / Triheptanoin
Triheptanoin
also known as
UX007 · C7 oil · Dermofeel tc 7 · Dojolvi · Dub thg · Heptanoin, tri- · Lanol 37 t · Radiamuls 2375 · +6 more
Regulatory milestones approvals, filings & regulatory actions · 2 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Filed disorder of fatty acid oxidation and ketogenesis 1 milestone | |||||
| Filed | US (FDA) | — | disorder of fatty acid oxidation and ketogenesis | 2019-08-01 | “submitted to the U.S. Food and Drug Administration ( FDA ) a New Drug Application (NDA) for UX007 (triheptanoin)” ultragenyx.com ↗ |
| Approved Indication not stated 1 milestone | |||||
| Approved | US (FDA) | DOJOLVI | — | 2020-06-30 | fda.gov ↗ |
Trials 31
News releases announcing trial results or a regulatory action · 22
| Date | Issuer | Release |
|---|---|---|
| 2021-08-23 | Ultragenyx Pharmaceutical Inc | Regulatory Ultragenyx Announces Approval of Dojolvi® (triheptanoin) in Brazil for the Treatment of Long-chain Fatty Acid Oxidation Disorders in Adults and Children ultragenyx.com ↗
This approval of Dojolvi is critical to providing therapeutic options for patients in Brazil and we are now working with urgency to obtain reimbursement approval in order to support broad access to therapy. |
| 2021-02-17 | Ultragenyx Pharmaceutical Inc | Regulatory Ultragenyx Announces Approval of Dojolvi™ (UX007/triheptanoin) in Canada for the Treatment of Long-chain Fatty Acid Oxidation Disorders in Adults and Children ultragenyx.com ↗
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare genetic diseases, today announced that Health Canada has approved Dojolvi™ (triheptanoin) as a source of calories and fatty acids for the treatment of adult and pediatric patients with long-chain fatty acid oxidation disorders (LC-FAOD). |
| 2020-06-30 | Ultragenyx Pharmaceutical Inc | Regulatory Ultragenyx Announces U.S. FDA Approval of Dojolvi™ (UX007/triheptanoin), the First FDA-Approved Therapy for the Treatment of Long-chain Fatty Acid Oxidation Disorders ultragenyx.com ↗
today announced that the U.S. Food and Drug Administration (FDA) has approved Dojolvi™ (triheptanoin) as a source of calories and fatty acids for the treatment of pediatric and adult patients with molecularly confirmed long-chain fatty acid oxidation disorders (LC-FAOD). |
| 2019-11-05 | Ultragenyx Pharmaceutical Inc | Regulatory Ultragenyx Reports Third Quarter 2019 Financial Results and Corporate Update ultragenyx.com ↗
The U.S. FDA accepted for review the New Drug Application (NDA) and has set a Prescription Drug User Fee Act (PDUFA) date of July 31, 2020. |
| 2019-10-14 | Ultragenyx Pharmaceutical Inc | Regulatory Ultragenyx Announces FDA Accepts New Drug Application for UX007 (triheptanoin) for Treatment of Long-chain Fatty Acid Oxidation Disorders ultragenyx.com ↗
today announced that the U.S. Food and Drug Administration ( FDA ) has accepted for review the company’s New Drug Application (NDA) for UX007 (triheptanoin) for the treatment of long-chain fatty acid oxidation disorders (LC-FAOD), |
| 2019-08-01 | Ultragenyx Pharmaceutical Inc | Regulatory Ultragenyx Reports Second Quarter 2019 Financial Results and Corporate Update ultragenyx.com ↗
Ultragenyx submitted a New Drug Application (NDA) to the FDA earlier this week. |
| 2019-08-01 | Ultragenyx Pharmaceutical Inc | Regulatory Ultragenyx Announces Submission of New Drug Application to FDA for UX007 (triheptanoin) for Treatment of Long-Chain Fatty Acid Oxidation Disorders ultragenyx.com ↗
The submission of this NDA is an important step toward providing a new treatment option and we look forward to working with the FDA on this review. |
| 2019-04-16 | Ultragenyx Pharmaceutical Inc | Regulatory Ultragenyx Announces UX007 Granted Fast Track Designation and Rare Pediatric Disease Designation by U.S. FDA for Treatment of Long-Chain Fatty Acid Oxidation Disorders ultragenyx.com ↗ |
| 2019-01-22 | Ultragenyx Pharmaceutical Inc | Results Ultragenyx Announces Positive Topline Data from Ongoing Long-Term Extension Study of UX007 for the Treatment of Long-chain Fatty Acid Oxidation Disorders ultragenyx.com ↗ |
| 2018-10-26 | Ultragenyx Pharmaceutical Inc | Results Ultragenyx Announces Negative Topline Results from Phase 3 Study of UX007 in Patients with Glut1 DS with Disabling Movement Disorders ultragenyx.com ↗
The study also did not meet its key secondary endpoints. |
| 2018-08-29 | Ultragenyx Pharmaceutical Inc | Regulatory Ultragenyx Announces FDA Accepts Proposal to Submit an NDA for UX007 for the Treatment of Long-Chain Fatty Acid Oxidation Disorders ultragenyx.com ↗
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced that the U.S. Food and Drug Administration ( FDA ) has accepted Ultragenyx’s most recent proposal to submit a New Drug Application (NDA) for UX007 for the treatment of long-chain fatty acid oxidation disorders (LC-FAOD) based on existing data. |
| 2017-03-22 | Ultragenyx Pharmaceutical Inc | Results Ultragenyx Announces Topline Data from Phase 2 UX007 Glucose Transporter Type-1 Deficiency Syndrome Seizure Study ultragenyx.com ↗
The study did not meet the primary endpoint of reducing the frequency of total number of observable and absence seizures among patients treated from baseline to Week 8 with UX007 compared to placebo. |
| 2016-11-30 | Ultragenyx Pharmaceutical Inc | Results Ultragenyx Announces Positive Data from Phase 2 Study of UX007 in Long-Chain Fatty Acid Oxidation Disorder Patients ultragenyx.com ↗
Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced positive 78-week data from the Phase 2 study of UX007 (triheptanoin) in patients with long-chain fatty acid oxidation disorder (LC-FAOD). |
| 2015-10-13 | Ultragenyx Pharmaceutical Inc | Results Ultragenyx Announces Positive Interim Data From Phase 2 Study of UX007 in Long-Chain Fatty Acid Oxidation Disorder Patients ultragenyx.com ↗
We are encouraged by these positive exercise tolerance and safety interim results, which add to the growing body of data that support the continued development of UX007 in LC-FAOD," said Sunil Agarwal, M.D., Chief Medical Officer of Ultragenyx. |
| 2015-09-01 | Ultragenyx Pharmaceutical Inc | Results Ultragenyx Announces Presentation of Data From Long-Chain Fatty Acid Oxidation Disorder Patients With Cardiomyopathy Treated With Triheptanoin ultragenyx.com ↗
All patients demonstrated an improvement in ejection fraction (EF), a measure of cardiac function evaluated by echocardiogram, after treatment with triheptanoin. |
| 2015-05-11 | Ultragenyx Pharmaceutical Inc | Results Ultragenyx Reports First Quarter 2015 Financial Results and Corporate Update ultragenyx.com ↗
Treatment with triheptanoin resulted in a 90% reduction in the number of paroxysmal motor events in six patients with movement disorders associated with Glut1 DS (p=0.028). |
| 2015-04-23 | Ultragenyx Pharmaceutical Inc | Regulatory Ultragenyx Granted Additional Orphan Drug Designations for Triheptanoin ultragenyx.com ↗
Ultragenyx Pharmaceutical Inc. (Nasdaq:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced that it has received two orphan drug designations for triheptanoin (UX007). |
| 2015-04-22 | Ultragenyx Pharmaceutical Inc | Results Ultragenyx Announces Positive Data From Investigator-Sponsored Trial of Triheptanoin in Glucose Transporter Type-1 Deficiency Syndrome ultragenyx.com ↗
The positive data presented at AAN have prompted us to expand our development efforts for triheptanoin in Glut1 deficiency to include a new study focused on movement disorders, in addition to the ongoing study in seizures. |
| 2014-10-27 | Ultragenyx Pharmaceutical Inc | Regulatory Ultragenyx Granted Orphan Drug Designation for Triheptanoin for the Treatment of Glucose Transporter Type-1 Deficiency Syndrome ultragenyx.com ↗
The FDA's decision to grant triheptanoin orphan drug designation for Glut1 deficiency syndrome is another important step in our plan to help treat patients with this disease, |
| 2013-12-17 | Ultragenyx Pharmaceutical Inc | Regulatory Ultragenyx Investigational New Drug Application for Triheptanoin for the Treatment of Glucose Transporter Type-1 Deficiency Syndrome is in Effect ultragenyx.com ↗
Ultragenyx Investigational New Drug Application for Triheptanoin for the Treatment of Glucose Transporter Type-1 Deficiency Syndrome is in Effect |
| 2013-09-16 | Ultragenyx Pharmaceutical Inc | Results Ultragenyx Announces Positive Data from Retrospective Study of UX007 Triheptanoin in Patients with Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD) ultragenyx.com ↗
Ultragenyx Announces Positive Data from Retrospective Study of UX007 Triheptanoin in Patients with Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD) |
| 2013-08-26 | Ultragenyx Pharmaceutical Inc | Regulatory Ultragenyx Investigational New Drug Application for UX007 is Active ultragenyx.com ↗
Ultragenyx Investigational New Drug Application for UX007 is Active |
All press releases naming this drug 34 releases
Evidence & citations 18 cited values
Every value below carries the sentence it was read from. 37 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | Triheptanoin | “The purpose of this project is to study the efficacy of triheptanoin oil in patients with GLUT1 deficiency syndrome.” NCT02014883 ↗ |
| Known as | C7 oil | “Triheptanoin (C7 oil) is a triglyceride of the anaplerotic odd-chain fatty acid heptonate.” NCT02000960 ↗ |
| Known as | Dermofeel tc 7 | ChEMBL registry synonym — accepted as the source's own label CHEMBL4297585 ↗ |
| Known as | Dojolvi | ChEMBL registry synonym — accepted as the source's own label CHEMBL4297585 ↗ |
| Known as | Dub thg | ChEMBL registry synonym — accepted as the source's own label CHEMBL4297585 ↗ |
| Known as | Heptanoin, tri- | ChEMBL registry synonym — accepted as the source's own label CHEMBL4297585 ↗ |
| Known as | Lanol 37 t | ChEMBL registry synonym — accepted as the source's own label CHEMBL4297585 ↗ |
| Known as | Radiamuls 2375 | ChEMBL registry synonym — accepted as the source's own label CHEMBL4297585 ↗ |
| Known as | SpezialölÒ 107 | “Triheptanoin (SpezialölÒ 107®)” NCT02201368 ↗ |
| Known as | Trienanthoin | ChEMBL registry synonym — accepted as the source's own label CHEMBL4297585 ↗ |
| Known as | Triheptanoic glyceride | ChEMBL registry synonym — accepted as the source's own label CHEMBL4297585 ↗ |
| Known as | Triheptanoina | ChEMBL registry synonym — accepted as the source's own label CHEMBL4297585 ↗ |
| Known as | Triheptanoine | ChEMBL registry synonym — accepted as the source's own label CHEMBL4297585 ↗ |
| Known as | Trioenanthoin | ChEMBL registry synonym — accepted as the source's own label CHEMBL4297585 ↗ |
| Known as | UX007 | “This treatment plan is intended for the use of UX007 (triheptanoin) in the treatment of a single patient with Glucose Transporter 1 (GLUT1) Deficiency Syndrome.” NCT02968953 ↗6“Triheptanoin (UX007) is a medium chain triglyceride of three seven-carbon fatty acids (C7), on a glycerol backbone, with a molecular formula of C24H44O6.” NCT02500082 ↗ |
| Action | Restore | “The hypothesis is that triheptanoin will restore deficient energy metabolism in these patients” NCT02500082 ↗ |
| Modality | Other / unclassified | “triheptanoin, a 7 carbon triglyceride” NCT00947960 ↗ |
| Route | Oral | “The trial involves 18 subjects ingesting a diet supplemented with triheptanoin” NCT00947960 ↗ |