TRIHEPTANOIN - Generic Drug Details

What are the generic sources for triheptanoin and what is the scope of patent protection?

Triheptanoin is the generic ingredient in one branded drug marketed by Ultragenyx Pharm Inc and is included in one NDA. There are two patents protecting this compound. Additional information is available in the individual branded drug profile pages.

Triheptanoin has eight patent family members in four countries.

There is one drug master file entry for triheptanoin. One supplier is listed for this compound.

Summary for TRIHEPTANOIN

International Patents:	8
US Patents:	2
Tradenames:	1
Applicants:	1
NDAs:	1
Drug Master File Entries:	1
Finished Product Suppliers / Packagers:	1
Raw Ingredient (Bulk) Api Vendors:	64
Clinical Trials:	37
What excipients (inactive ingredients) are in TRIHEPTANOIN?	TRIHEPTANOIN excipients list
DailyMed Link:	TRIHEPTANOIN at DailyMed

DrugPatentWatch^® Estimated Loss of Exclusivity (LOE) Date for TRIHEPTANOIN

Constraining patent/regulatory exclusivity:

INDICATED 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)

Dosage:

LIQUID;ORAL

^*The generic entry opportunity date is the latter of the last compound-claiming patent and the last regulatory exclusivity protection. Many factors can influence early or later generic entry. This date is provided as a rough estimate of generic entry potential and should not be used as an independent source.

Recent Clinical Trials for TRIHEPTANOIN

Identify potential brand extensions & 505(b)(2) entrants

Sponsor	Phase
Jerry Vockley, MD, PhD	PHASE2
Ultragenyx Pharmaceutical Inc	PHASE2
Jerry Vockley, MD, PhD	Phase 2

See all TRIHEPTANOIN clinical trials

Pharmacology for TRIHEPTANOIN

Drug Class

Medium-chain Triglyceride

Anatomical Therapeutic Chemical (ATC) Classes for TRIHEPTANOIN

A16AX	Various alimentary tract and metabolism products
A16A	OTHER ALIMENTARY TRACT AND METABOLISM PRODUCTS
A16	OTHER ALIMENTARY TRACT AND METABOLISM PRODUCTS
A	Alimentary tract and metabolism

Paragraph IV (Patent) Challenges for TRIHEPTANOIN

Tradename	Dosage	Ingredient	Strength	NDA	ANDAs Submitted	Submissiondate
DOJOLVI	Oral Liquid	triheptanoin	100% w/w	213687	3	2024-07-01

US Patents and Regulatory Information for TRIHEPTANOIN

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	TE	Type	RLD	RS	Patent No.	Patent Expiration	Product	Substance	Delist Req.	Exclusivity Expiration
Ultragenyx Pharm Inc	DOJOLVI	triheptanoin	LIQUID;ORAL	213687-001	Jun 30, 2020		RX	Yes	Yes
Ultragenyx Pharm Inc	DOJOLVI	triheptanoin	LIQUID;ORAL	213687-001	Jun 30, 2020		RX	Yes	Yes			Y
Ultragenyx Pharm Inc	DOJOLVI	triheptanoin	LIQUID;ORAL	213687-001	Jun 30, 2020		RX	Yes	Yes
Ultragenyx Pharm Inc	DOJOLVI	triheptanoin	LIQUID;ORAL	213687-001	Jun 30, 2020		RX	Yes	Yes			Y

Expired US Patents for TRIHEPTANOIN

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	Patent No.	Patent Expiration
Ultragenyx Pharm Inc	DOJOLVI	triheptanoin	LIQUID;ORAL	213687-001	Jun 30, 2020

International Patents for TRIHEPTANOIN

Country	Patent Number	Title	Estimated Expiration
Canada	2573054	METHODE DE TRAITEMENT D'UNE MALADIE DE SURCHARGE DUE A L'ACCUMULATION DE GLYCOGENES OU DE POLYSACCHARIDES (GLYCOGEN OR POLYSACCHARIDE STORAGE DISEASE TREATMENT METHOD)
World Intellectual Property Organization (WIPO)	2006014353
European Patent Office	3300727	PROCEDES POUR LE TRAITEMENT DE GLYCOGÉNOSES ET DE MALADIES DU STOCKAGE DES POLYSACCHARIDES (GLYCOGEN OR POLYSACCHARIDE STORAGE DISEASE TREATMENT METHOD)
Mexico	2007000304	METODO DE TRATAMIENTO DE ENFERMEDAD DE ALMACENAMIENTO DE GLUCOGENO O POLISACARIDOS. (GLYCOGEN OR POLYSACCHARIDE STORAGE DISEASE TREATMENT METHOD.)
Mexico	338247	METODO DE TRATAMIENTO DE ENFERMEDAD DE ALMACENAMIENTO DE GLUCOGENO O POLISACARIDOS. (GLYCOGEN OR POLYSACCHARIDE STORAGE DISEASE TREATMENT METHOD.)
Canada	2784420	METHODE DE TRAITEMENT D'UNE MALADIE DE SURCHARGE DUE A L'ACCUMULATION DE GLYCOGENES OU DE POLYSACCHARIDES (GLYCOGEN OR POLYSACCHARIDE STORAGE DISEASE TREATMENT METHOD)
European Patent Office	1773317	METHODE DE TRAITEMENT D'UNE MALADIE DE SURCHARGE DUE A L'ACCUMULATION DE GLYCOGENES OU DE POLYSACCHARIDES (GLYCOGEN OR POLYSACCHARIDE STORAGE DISEASE TREATMENT METHOD)

Market Dynamics and Financial Trajectory for Triheptanoin

Last updated: July 27, 2025

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Introduction

Triheptanoin, a triglyceride composed of three seven-carbon fatty acids, operates primarily as an anaplerotic agent targeting rare metabolic disorders. Its unique mechanism replenishes the tricarboxylic acid (TCA) cycle, offering therapeutic potential in conditions such as long-chain fatty acid oxidation deficiencies, certain epilepsies, and other metabolic diseases. This report analyzes current market dynamics, key drivers and barriers, and forecasts the financial trajectory for triheptanoin over the next decade.

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Regulatory Status and Market Entry

Triheptanoin's regulatory journey has been marked by limited but focused approvals. Notably, it was approved by the FDA in 2016 under the name UX007 for the treatment of recurrent infantile encephalopathy and primary hyperlipidemia associated with long-chain fat oxidation disorders [1]. This accelerated approval facilitated its entry into niche markets addressing ultra-rare conditions.

Market penetration is constrained due to its orphan drug designation, which benefits from incentives like market exclusivity, but also entails limited commercial scalability. Early-stage regulatory approvals in Europe and other jurisdictions remain pending or exploratory, with clinical trials ongoing (e.g., for epileptic syndromes like Glutaric Aciduria Type I).

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Market Drivers

1. Unmet Medical Needs in Rare Metabolic Disorders
Triheptanoin addresses critical gaps for patients with metabolic deficiencies unresponsive to conventional therapy. The lack of effective treatments and the grave prognosis associated with these disorders propel demand, particularly among pediatric populations.

2. Orphan Drug Incentives
Regulatory stipulations favoring orphan drugs—such as patent extensions, tax credits, and priority review—encourage pharmaceutical investment in triheptanoin development. These incentives mitigate the economic risk associated with small patient populations.

3. Expanding Clinical Evidence
Preclinical and early clinical data support triheptanoin's efficacy in certain genetic epilepsies and neurodegenerative disorders. Promising trial outcomes could extend indications, broadening addressable markets [2].

4. Increasing Awareness of Metabolic Disorders
Enhanced recognition, facilitated by improved screening programs and genetic diagnostics, elevates diagnosis rates. This trend naturally expands the pool of potential patients eligible for triheptanoin therapy.

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Market Barriers

1. Limited Market Size and High Cost
The ultra-rare nature of target conditions yields a small patient base, restricting revenue opportunities. The high development costs for orphan drugs translate into premium pricing, which can limit payer acceptance and reimbursement.

2. Competitive Landscape
While triheptanoin currently faces minimal direct competition, emerging treatments based on gene therapy or alternative metabolic approaches (e.g., mRNA-based therapies) threaten future market share.

3. Clinical and Manufacturing Challenges
The complex synthesis of triheptanoin and required cold chain logistics elevate manufacturing costs. Moreover, the limited number of clinical trials might slow broader approval and acceptance.

4. Regulatory and Reimbursement Hurdles
Access barriers continue due to variable reimbursement policies across regions, especially in countries with limited orphan drug frameworks. Regulatory authorities may demand extensive evidence before broad approval.

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Financial Trajectory and Market Forecasts

Current Valuation and Revenue
As of 2023, triheptanoin's global sales are estimated at under USD 50 million, predominantly driven by initial indications and compassionate use cases [3]. Its revenue remains marginal, reflecting early commercialization phases.

Projections and Growth Potential
By leveraging current trends—such as expanding diagnosis, ongoing clinical trials, and potential new indications—the market is poised for moderate growth. Analyst estimates project a compound annual growth rate (CAGR) of approximately 8-10% over the next five years, driven by increased approval and adoption in emerging indications [4].

Key Factors Influencing Growth

• Expansion of approved indications: Positive trial outcomes in epileptic disorders could unlock new revenue streams.
• Geographic expansion: Approvals in Europe, Asia, and Latin America could multiply market access.
• Pricing and reimbursement strategies: High-value contracts with payers could improve margins, although reimbursement policies for ultra-rare drugs often challenge profitability.

Long-term Outlook
Over ten years, financial growth relies on further clinical validation, regulatory approvals, and market acceptance. If triheptanoin successfully demonstrates efficacy in broader metabolic and neurological conditions, revenues could approach USD 100-200 million globally, considering cumulative sales and new markets.

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Strategic Considerations

• Partnerships or Licensing Agreements: Collaborations with biotech firms could accelerate clinical development and geographical expansion.
• Investment in Clinical Trials: Prioritizing studies in epilepsy, neurodegenerative diseases, and other rare disorders can diversify revenue streams.
• Cost Management: Streamlining manufacturing and supply chain processes will be crucial to maintaining margins given high R&D and production costs.

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Conclusion

Triheptanoin occupies a niche rather than a mass-market segment due to its orphan drug status and specific therapeutic applications. Its future financial trajectory hinges on successful regulatory approvals for additional indications, geographic expansion, and clinical validation. While current revenues remain modest, strategic moves in clinical development and partnership formations could catalyze sustainable growth.

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Key Takeaways

• Triheptanoin’s market is driven primarily by its role in rare metabolic disorders with high unmet needs.
• Regulatory incentives favor its development, but small patient populations limit revenue potential.
• Clinical trial progress and expanding indications are critical to market growth; current growth estimates forecast modest but steady expansion.
• Manufacturing and reimbursement challenges must be addressed to optimize profitability.
• Long-term success depends on innovation in clinical applications and strategic collaborations.

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FAQs

Q1: What are the primary indications for triheptanoin currently?
A: Its primary approved use is for treating long-chain fatty acid oxidation disorders, such as recurrent infantile encephalopathy and hyperlipidemia associated with energy deficiency.

Q2: How does triheptanoin compare to other metabolic therapies?
A: Triheptanoin’s unique anaplerotic mechanism distinguishes it from other treatments that primarily manage symptoms; it aims to correct underlying metabolic deficiencies.

Q3: What are the main challenges to expanding triheptanoin’s market?
A: Challenges include its small target population, high treatment costs, limited clinical data for broader indications, and reimbursement complexities.

Q4: Are there ongoing clinical trials that could expand triheptanoin's approved uses?
A: Yes, trials are underway exploring its efficacy in epileptic syndromes like Glutaric Aciduria Type I and neurodegenerative conditions, which could widen its application.

Q5: What is the outlook for triheptanoin’s future market growth?
A: With continued clinical success and regulatory approvals, the market is projected to grow moderately over the next decade, contingent upon expanded indications and geographic reach.

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Sources

[1] U.S. Food and Drug Administration. (2016). FDA approval of UX007 for treating certain metabolic disorders.

[2] ClinicalTrials.gov. (2022). Active studies exploring triheptanoin in neurological and metabolic diseases.

[3] MarketWatch. (2023). Financial performance analysis and sales estimates for triheptanoin.

[4] GlobalData. (2022). Forecast reports on orphan drug markets and future growth prospects.