Triheptanoin

Phase 3

Ultragenyx Pharmaceutical Inc. (RARE)

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What is LOA?

Phase 3

Development Phase

Upcoming Catalysts

Historical Events

Regulatory Approvals

Upcoming Catalysts

What are catalysts?

Aug '27

RAREPhase 3$1.5B

Triheptanoin Phase 3 Est. Readout

Triheptanoin• Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)69

Aug 2027~Confirmed

CT.gov

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Competitive Landscape

2 competitors in Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)

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Drug Information

Target Indication

Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)

Clinical Trial

NCT05933200

Last updated: 12/4/2025

Patent & Exclusivity

No patent data available. Pre-approval drugs may not have Orange Book listings.

Source: FDA Orange Book

Drug Science

Mechanism of action & safety profile

Developer

RARE

Ultragenyx Pharmaceutical Inc.

Pharmaceutical Preparations•North America•NASDAQ

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