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CLINICAL TRIALS PROFILE FOR DOJOLVI

All Clinical Trials for dojolvi

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT05933200 ↗	A Study to Determine the Effect of Triheptanoin Compared With Even-chain, MCT on MCEs in Pediatric Patients With LC-FAOD	Recruiting	Ultragenyx Pharmaceutical Inc	Phase 3	2023-02-28	The main goal of this study is to evaluate the effects of triheptanoin versus Medium-chain Triglycerides (MCT) on frequency of Major Clinical Events (MCEs).
NCT06067802 ↗	Study of Triheptanoin for the Prevention of Hypoglycemia in Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD)	Recruiting	Ultragenyx Pharmaceutical Inc	Phase 2	2024-08-01	This is a medical research study to test a medication in adult patients with a disease called medium-chain acyl-CoA dehydrogenase deficiency (MCADD). The medication is triheptanoin, which is currently FDA approved for the treatment of Long-Chain Fatty Acid Oxidation Disorders. Previous research suggests that triheptanoin may also be effective in the treatment MCADD. This study will investigate the safety and efficacy (how well it works) of triheptanoin in patients with MCADD.
NCT06067802 ↗	Study of Triheptanoin for the Prevention of Hypoglycemia in Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD)	Recruiting	Jerry Vockley, MD, PhD	Phase 2	2024-08-01	This is a medical research study to test a medication in adult patients with a disease called medium-chain acyl-CoA dehydrogenase deficiency (MCADD). The medication is triheptanoin, which is currently FDA approved for the treatment of Long-Chain Fatty Acid Oxidation Disorders. Previous research suggests that triheptanoin may also be effective in the treatment MCADD. This study will investigate the safety and efficacy (how well it works) of triheptanoin in patients with MCADD.

Clinical Trial Conditions for dojolvi

Condition Name

Condition Name for dojolvi
Intervention	Trials
Medium-chain Acyl-CoA Dehydrogenase Deficiency	1
Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)	1

Condition MeSH

Condition MeSH for dojolvi
Intervention	Trials
Hypoglycemia	1
Lipid Metabolism, Inborn Errors	1

Clinical Trial Locations for dojolvi

Trials by Country

Trials by Country for dojolvi
Location	Trials
Poland	3
Spain	2
Czechia	1
Saudi Arabia	1
United Kingdom	1

Trials by US State

Trials by US State for dojolvi
Location	Trials
Pennsylvania	1

Clinical Trial Progress for dojolvi

Clinical Trial Phase

Clinical Trial Phase for dojolvi
Clinical Trial Phase	Trials
Phase 3	1
Phase 2	1

Clinical Trial Status

Clinical Trial Status for dojolvi
Clinical Trial Phase	Trials
Recruiting	2

Clinical Trial Sponsors for dojolvi

Sponsor Name

Sponsor Name for dojolvi
Sponsor	Trials
Ultragenyx Pharmaceutical Inc	2
Jerry Vockley, MD, PhD	1

Sponsor Type

Sponsor Type for dojolvi
Sponsor	Trials
Industry	2
Other	1

Dojolvi Market Analysis and Financial Projection

DOJOLVI: A Breakthrough in Treating Long-Chain Fatty Acid Oxidation Disorders

Introduction to DOJOLVI

DOJOLVI, also known as triheptanoin, is a groundbreaking medication approved by the U.S. Food and Drug Administration (FDA) on June 30, 2020, for the treatment of pediatric and adult patients with long-chain fatty acid oxidation disorders (LC-FAOD)[3][4][5].

Clinical Trials Overview

The FDA approval of DOJOLVI was based on evidence from three clinical trials: Trial 1, Trial 2, and Trial 3.

Trial Design and Participants

• Trials 1 and 2 were conducted to evaluate the safety of DOJOLVI and enrolled pediatric and adult patients with LC-FAOD at 11 sites in the United States and the United Kingdom. Trial 1 involved patients receiving DOJOLVI for 78 weeks, while Trial 2, which is ongoing, includes patients who were previously treated with DOJOLVI and those who were not[1].
• Trial 3 was a 4-month double-blind, randomized, controlled trial that compared triheptanoin (DOJOLVI) with trioctanoin. This trial enrolled patients with a confirmed diagnosis of LC-FAOD and assessed cardiac function and metabolic stability[1].

Efficacy and Safety Outcomes

• The efficacy of DOJOLVI was demonstrated in Trial 3, where patients receiving DOJOLVI showed similar heart function to those receiving trioctanoin after four months of treatment. Baseline cardiovascular function was normal in both groups, and this remained consistent after the treatment period[1].
• The most common adverse reactions reported in the pooled safety population of Trials 1 and 2 were gastrointestinal-related, including abdominal pain, diarrhea, vomiting, and nausea[1].

Market Analysis

Market Launch and Availability

• Following FDA approval, Ultragenyx Pharmaceutical Inc. launched DOJOLVI in the U.S. market, making it the first approved therapy for LC-FAOD. The company has been working to ensure access to the drug for all eligible patients in the U.S.[3][4].

Pricing and Revenue Projections

• DOJOLVI is priced at $4,875 per vial, translating to an average net price of $138,000 per patient per year. According to investment bank Piper Sandler, the drug is projected to earn $270 million in sales by fiscal year 2025[5].

Market Impact

• The approval of DOJOLVI marks a significant milestone for patients with LC-FAOD, a group of rare, lifelong, and life-threatening genetic disorders. This drug provides hope for patients who previously had limited treatment options. The genetic condition affects between 2,000 and 3,500 children and adults in the U.S. and up to 14,000 patients in the developed world[5].

Market Projections and Future Developments

Revenue Expectations

• Ultragenyx expects DOJOLVI revenue to be in the range of $75 million to $80 million. The company's financial performance, including product sales in Latin America and Turkey, and royalties in Europe and North America, indicates a strong market presence[2].

Clinical Milestones and Future Studies

• Ultragenyx is on track to initiate the pivotal Phase 3 Aspire study by the end of 2024 and the Aurora study in 2025. Additionally, the company has received a positive assessment from Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) for Conditional Early Approval, with plans to file a Japanese New Drug Application (J-NDA) in mid-2025[2].

Industry and Patient Impact

Patient Benefits

• DOJOLVI is designed to correct the metabolic imbalance in patients with LC-FAOD by providing fatty acids for energy and replacing metabolites. This treatment offers a new tool for clinicians to manage the disease, reducing the frequency of hospitalizations and major medical events[4][5].

Expert Insights

• "FDA approval of Dojolvi for the treatment of long-chain fatty acid oxidation disorders is a huge milestone for patients and their families, as it is the first such medication," said Dr. Jerry Vockley, Professor of Human Genetics and Chief of Medical Genetics at Children's Hospital of Pittsburgh of UPMC. "Rare disease research, especially development of new therapies, is an arduous and time-consuming process, and today’s announcement represents the culmination of nearly 20 years of work on the part of numerous researchers, clinicians, and patients."[4]

Key Takeaways

• First FDA-Approved Therapy: DOJOLVI is the first FDA-approved treatment for LC-FAOD, a group of rare genetic disorders.
• Clinical Trials: The drug was approved based on three clinical trials, with Trial 3 demonstrating similar heart function in patients receiving DOJOLVI compared to those receiving trioctanoin.
• Market Availability: DOJOLVI is commercially available in the U.S. market, with Ultragenyx working to ensure patient access.
• Revenue Projections: The drug is expected to generate significant revenue, with projections of $270 million in sales by fiscal year 2025.
• Future Developments: Ultragenyx is planning additional studies and has received positive feedback for Conditional Early Approval in Japan.

FAQs

1. What is DOJOLVI used for?

   • DOJOLVI is used as a source of calories and fatty acids for the treatment of pediatric and adult patients with long-chain fatty acid oxidation disorders (LC-FAOD)[3][4].

2. What are the common side effects of DOJOLVI?

   • The most common adverse reactions include abdominal pain, diarrhea, vomiting, and nausea[1].

3. How was the efficacy of DOJOLVI evaluated?

   • The efficacy was evaluated in a 4-month double-blind, randomized, controlled trial (Trial 3) comparing triheptanoin (DOJOLVI) with trioctanoin[1].

4. What is the pricing of DOJOLVI?

   • DOJOLVI is priced at $4,875 per vial, with an average net price of $138,000 per patient per year[5].

5. What are the future clinical milestones for DOJOLVI?

   • Ultragenyx plans to initiate the Phase 3 Aspire study by the end of 2024 and the Aurora study in 2025, and will file a J-NDA in Japan in mid-2025[2].

Sources

1. FDA Drug Trials Snapshots: DOJOLVI - FDA
2. Form 8-K for Ultragenyx Pharmaceutical INC - Ultragenyx
3. Ultragenyx Launches Dojolvi In Us Market To Treat Long-Chain Fatty Acid Oxidation Disorders - Clival
4. Ultragenyx Announces U.S. FDA Approval of Dojolvi™ (UX007/triheptanoin) - Ultragenyx
5. Ultragenyx wins early approval for rare disease drug - BioPharma Dive