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:	34
Patent Applications:	899
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	Phase 2
Ultragenyx Pharmaceutical Inc	Early Phase 1
Areeg El-Gharbawy	Early Phase 1

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

International Patents for TRIHEPTANOIN

Country	Patent Number	Title	Estimated Expiration
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)
Mexico	2007000304	METODO DE TRATAMIENTO DE ENFERMEDAD DE ALMACENAMIENTO DE GLUCOGENO O POLISACARIDOS. (GLYCOGEN OR POLYSACCHARIDE STORAGE DISEASE TREATMENT METHOD.)

TRIHEPTANOIN Market Analysis and Financial Projection Experimental

Market Dynamics and Financial Trajectory for Triheptanoin

Introduction

Triheptanoin, marketed under the brand name Dojolvi, is a drug that has garnered significant attention in the pharmaceutical industry, particularly for its use in treating rare metabolic disorders. Here, we delve into the market dynamics and financial trajectory of triheptanoin, highlighting its current status, growth prospects, and key factors influencing its market.

Market Segmentation

The triheptanoin market is segmented based on its type and application. The 94% Triglyceride of Heptanoic Acid segment has accounted for a noticeable share of the global triheptanoin market and is projected to experience significant growth in the near future[1].

Type

• 94% Triglyceride of Heptanoic Acid: This segment is expected to dominate the market due to its efficacy and widespread use.
• 95% Triglyceride of Heptanoic Acid: Although less dominant, this segment still holds a significant market share.

Application

• Medical Use: Triheptanoin is primarily used in the medical field, particularly for treating rare metabolic disorders such as long-chain fatty acid oxidation disorders (LC-FAOD) and glycogen storage disease type Ia (GSDIa). The medical use segment is expected to expand at a significant compound annual growth rate (CAGR) throughout the forecast period[1].

Market Drivers

Several factors are driving the growth of the triheptanoin market:

Regulatory Approvals

• Triheptanoin was granted orphan drug designation by the FDA in 2015, which has been a significant driver for its development and market approval[3].

Clinical Efficacy

• The drug has shown promising results in clinical trials, particularly in reducing major clinical events in patients with LC-FAOD and GSDIa. For instance, the randomized Gillingham trial provided adequate evidence to support its approval for a nutritional indication[3].

Increasing Demand

• The growing need for effective treatments for rare metabolic disorders is driving the demand for triheptanoin. Its use as a first-line therapy in selected patients, especially neonates or infants with acute, life-threatening conditions, further boosts its demand[4].

Market Restraints

Despite the positive outlook, there are several restraints that could impact the market growth:

Regulatory Challenges

• The initial review of the new drug application (NDA) for triheptanoin was marked by robust disagreement within the review team regarding the sufficiency of evidence for its efficacy. This led to recommendations for further randomized studies to compare its efficacy against control groups[3].

Competition

• The market for rare disease treatments is competitive, with other drugs and therapies vying for market share. This competition can affect the pricing and distribution strategies for triheptanoin.

Financial Trajectory

Revenue Growth

• The financial performance of companies involved in the production and distribution of triheptanoin, such as Ultragenyx Pharmaceutical, indicates strong revenue growth. For example, Ultragenyx reported total revenues of $139 million in Q3 2024, up 42% year-over-year, with Dojolvi revenue increasing by 29% to $21 million[2].

Market Size and Forecast

• The global triheptanoin market size in 2023 was significant, and it is expected to grow at a notable CAGR from 2024 to 2031. The market is segmented by region, with North America, Europe, Asia Pacific, South America, and the Middle East and Africa each contributing to the overall growth[1].

Key Players

• Companies like Stearinerie Dubois, Dr. Straetmans, Evonik, and Ultragenyx Pharmaceutical are key players in the triheptanoin market. These companies are focusing on strategy building and product portfolio expansion to strengthen their market position[1].

Regional Analysis

The triheptanoin market is analyzed at the global, regional, and country levels. Here are some key insights:

North America

• This region is expected to be a significant market for triheptanoin due to the presence of major pharmaceutical companies and a well-established healthcare infrastructure.

Europe

• Europe is another key market, driven by regulatory approvals and the increasing demand for rare disease treatments.

Asia Pacific

• The Asia Pacific region is expected to grow rapidly due to increasing healthcare spending and the growing awareness of rare metabolic disorders.

Technological and Regulatory Trends

Technological Advancements

• Advances in drug development and manufacturing processes are crucial for the triheptanoin market. For instance, the use of gene therapy and mRNA platforms by companies like Ultragenyx is expected to further enhance the market[5].

Regulatory Environment

• Environmental and safety regulations play a critical role in shaping the chemical and pharmaceutical industries. Compliance with these regulations is essential for the production, pricing, and distribution of triheptanoin[1].

Financial Performance of Key Companies

Ultragenyx Pharmaceutical

• Ultragenyx has shown strong financial performance, with total revenues increasing by 42% year-over-year. Despite reporting a net loss, the company has a strong cash position and reaffirmed its revenue guidance for 2024[2].

Key Takeaways

• Market Growth: The triheptanoin market is expected to grow significantly, driven by its efficacy in treating rare metabolic disorders.
• Regulatory Approvals: Orphan drug designation and approvals for specific indications are crucial for market growth.
• Financial Performance: Companies like Ultragenyx are reporting strong revenue growth and maintaining a robust financial position.
• Regional Analysis: North America, Europe, and the Asia Pacific are key regions driving the market growth.
• Technological Trends: Advances in drug development and manufacturing processes are expected to enhance the market.

FAQs

What is the primary use of triheptanoin in the medical field?

Triheptanoin is primarily used in the medical field for treating rare metabolic disorders such as long-chain fatty acid oxidation disorders (LC-FAOD) and glycogen storage disease type Ia (GSDIa).

Which segment dominates the triheptanoin market?

The 94% Triglyceride of Heptanoic Acid segment dominates the triheptanoin market and is expected to experience significant growth in the near future.

What are the key drivers for the triheptanoin market?

Key drivers include regulatory approvals, clinical efficacy, and increasing demand for effective treatments for rare metabolic disorders.

Which company is a major player in the triheptanoin market?

Ultragenyx Pharmaceutical is a major player in the triheptanoin market, with its product Dojolvi showing strong revenue growth.

What is the expected CAGR for the triheptanoin market from 2024 to 2031?

The global triheptanoin market is expected to grow at a notable CAGR from 2024 to 2031, although the exact CAGR figure is not specified in the available sources.

Sources

1. Cognitivemarketresearch.com: Triheptanoin Market Report 2024 (Global Edition)
2. Stocktitan.net: Ultragenyx Reports Third Quarter 2024 Financial Results and Corporate Update
3. Accessdata.fda.gov: 213687Orig1s000 - Integrated Review
4. Canjhealthtechnol.ca: Triheptanoin (Dojolvi) - Canadian Journal of Health Technologies
5. Ir.ultragenyx.com: J.P. Morgan Corporate Presentation - Ultragenyx Pharmaceutical Inc.