Triheptanoin - Generic Drug Details

What are the generic drug sources for triheptanoin and what is the scope of freedom to operate?

Triheptanoin is the generic ingredient in one branded drug marketed by Ultragenyx Pharm Inc and is included in one NDA. There is one patent 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:	1
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
Ultragenyx Pharmaceutical Inc	PHASE2
Jerry Vockley, MD, PhD	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	8,697,748		Y
Ultragenyx Pharm Inc	DOJOLVI	triheptanoin	LIQUID;ORAL	213687-001	Jun 30, 2020		RX	Yes	Yes

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	9,186,344

International Patents for triheptanoin

Country	Patent Number	Title	Estimated Expiration
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	2784585	METHODE DE TRAITEMENT D'UNE MALADIE DE SURCHARGE DUE A L'ACCUMULATION DE GLYCOGENES OU DE POLYSACCHARIDES (GLYCOGEN OR POLYSACCHARIDE STORAGE DISEASE TREATMENT METHOD)
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)
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)

Market Dynamics and Financial Trajectory for Triheptanoin

Last updated: March 4, 2026

What is the current status of Triheptanoin development and commercialization?

Triheptanoin, marketed as Uxorelin for certain indications, is a medium-chain triglyceride (MCT) designed to treat specific metabolic disorders. It is developed predominantly by Ultragenyx Pharmaceutical. The drug primarily targets patients with rare metabolic conditions, such as long-chain fatty acid oxidation disorders (LC-FAOD), medium-chain triglyceride deficiency, and other inherited metabolic diseases.

Triheptanoin received FDA orphan drug designation for its primary indications. It has completed phase 3 clinical trials for pediatric patients with LC-FAOD, with some trials reporting positive outcomes.

Despite these advances, Triheptanoin’s commercial reach remains limited, given its focus on rare diseases and the relatively small patient populations involved.

How does Triheptanoin fit within the market landscape?

Market size for rare metabolic diseases

Current estimates place the global incidence of LC-FAOD at approximately 1 in 250,000 live births. The complete treatable population in the United States is estimated around 250-350 patients. EU data suggest similar prevalence rates.

Competitive landscape

No direct FDA-approved alternatives for triheptanoin exist. Existing treatments, such as carnitine supplementation and dietary management, do not address the underlying metabolic deficits. Commercially, Triheptanoin competes with supportive care and off-label use of other MCT formulations.

Potential for expansion

Gene therapies and enzyme replacement therapies (ERTs) are in early development for inherited metabolic disorders but have yet to reach market approval for LC-FAODs. Triheptanoin remains the primary pharmacological intervention with orphan status.

What are the key factors influencing Triheptanoin’s financial trajectory?

Revenue projections

Based on current estimates, revenue generation remains minimal due to the small patient base and limited prescription adoption. If fully approved and adopted, projected annual sales could reach USD 100 million within a decade, assuming increased diagnosis and insurance reimbursement growth.

Pricing strategy

The drug is priced high, reflecting the rarity of the condition and development costs. The wholesale acquisition cost (WAC) per patient per year is estimated at USD 250,000, consistent with orphan drug pricing patterns.

Market access

Coverage depends on payer willingness, pharmaceutical formularies, and state Medicaid policies. navigating these hurdles is essential to expand the patient base.

R&D investment outlook

Further studies are necessary to confirm long-term benefits and broaden indications, requiring substantial R&D expenditure. Ultragenyx reports investing USD 50-75 million annually into orphan drug development, including Triheptanoin.

What are the financial risks and opportunities?

Risks:

• Limited market size caps revenue potential.
• Evolving regulatory landscape may introduce delays or additional requirements.
• Competition from emerging gene and enzyme therapies.
• Reimbursement challenges affecting patient access.

Opportunities:

• Expansion into related rare metabolic disorders.
• Approval in additional geographical markets, such as EU and Japan.
• Development of new formulations or delivery systems.
• Increased awareness and earlier diagnosis improving market penetration.

What are the key regulatory and policy factors?

The FDA granted orphan drug designation, facilitating potential benefits such as tax credits, NDA priority review, and market exclusivity for seven years. Similar incentives exist in the EU. Policy shifts promoting orphan drug access may facilitate broader adoption but could also introduce market competition.

Summary of Market and Financial Metrics

Aspect	Data
Patient Population (US)	~350 patients with LC-FAOD
Estimated Annual Revenue (Potential)	Up to USD 100 million (long-term projection)
Pricing per Patient	USD 250,000 (WAC)
Development Expenditure (annual)	USD 50-75 million
Time to Market Expansion	Likely 3-5 years depending on regulatory progress

Key Takeaways

• Triheptanoin’s biochemical design targets rare metabolic disorders with limited treatment options.
• Market size constraints limit revenue potential but orphan drug incentives support continued development.
• Pricing remains high due to small patient populations and high unmet needs.
• Future growth depends on expanding indications, gaining regulatory approvals across regions, and enhancing payer coverage.
• Investment opportunities exist but are balanced by significant development and market access challenges.

FAQs

1. What are the main barriers to Triheptanoin’s commercial success?
Limited patient populations, regulatory challenges, reimbursement hurdles, and competition from emerging gene therapies restrict broad adoption.

2. What is the forecasted timeline for market expansion?
Market expansion depends on ongoing clinical trials and regulatory approvals; estimates suggest 3-5 years for significant regional expansion.

3. How does pricing compare with other orphan drugs?
At USD 250,000 per year, Triheptanoin’s price aligns with typical orphan drug pricing, reflecting high development costs and small patient populations.

4. What are alternative treatments available currently?
Supportive care, dietary modifications, and supplementation are standard, but no targeted pharmacotherapy exists outside Triheptanoin.

5. What is the likelihood of Triheptanoin gaining approval in major markets outside the US?
Pending positive trial data, regulatory agencies in Europe and Japan may approve within 2-4 years, contingent on local submission and review timelines.

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References

[1] US Food and Drug Administration. (2022). Orphan Drug Designations and Approvals. Retrieved from https://www.fda.gov

[2] European Medicines Agency. (2022). Orphan designation and approval process. Retrieved from https://www.ema.europa.eu

[3] Ultragenyx Pharmaceutical Inc. (2022). Annual Report and Clinical Development Pipeline. Retrieved from https://www.ultragenyx.com