

Year
DEALS // DEV.
Country
Therapeutic Area
Study Phase
Deal Type
Product Type
Dosage Form
Lead Product
Target

Lead Product(s) : Triheptanoin
Therapeutic Area : Genetic Disease
Study Phase : Phase II
Sponsor : Ultragenyx Pharmaceutical
Deal Size : Inapplicable
Deal Type : Inapplicable
Study of Triheptanoin Use in Patients with Medium-Chain Acyl-CoA Dehydrogenase Deficiency
Details : Triheptanoin is a Other Small Molecule drug candidate, which is currently being evaluated in Phase II clinical studies for the treatment of Medium chain acyl CoA dehydrogenase Deficiency.
Product Name : Undisclosed
Product Type : Miscellaneous
Upfront Cash : Inapplicable
July 31, 2025
Lead Product(s) : Triheptanoin
Therapeutic Area : Genetic Disease
Highest Development Status : Phase II
Sponsor : Ultragenyx Pharmaceutical
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : Triheptanoin
Therapeutic Area : Rare Diseases and Disorders
Study Phase : Phase I
Sponsor : Jirair Krikor Bedoyan
Deal Size : Inapplicable
Deal Type : Inapplicable
Triheptanoin for Children with Primary-Specific Pyruvate Dehydrogenase Complex (PDC) Deficiency
Details : Triheptanoin is a Other Small Molecule drug candidate, which is currently being evaluated in phase I clinical studies for the treatment of Pyruvate Dehydrogenase Complex Deficiency Disease.
Product Name : Undisclosed
Product Type : Miscellaneous
Upfront Cash : Inapplicable
April 01, 2024
Lead Product(s) : Triheptanoin
Therapeutic Area : Rare Diseases and Disorders
Highest Development Status : Phase I
Sponsor : Jirair Krikor Bedoyan
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : Triheptanoin
Therapeutic Area : Genetic Disease
Study Phase : Phase II
Sponsor : Jerry Vockley
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : Triheptanoin is a Other Small Molecule drug candidate, which is currently being evaluated in phase II clinical studies for the treatment of Medium chain acyl CoA dehydrogenase Deficiency.
Product Name : Undisclosed
Product Type : Miscellaneous
Upfront Cash : Inapplicable
October 05, 2023
Lead Product(s) : Triheptanoin
Therapeutic Area : Genetic Disease
Highest Development Status : Phase II
Sponsor : Jerry Vockley
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : Triheptanoin
Therapeutic Area : Genetic Disease
Study Phase : Phase III
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : Triheptanoin is a Other Small Molecule drug candidate, which is currently being evaluated in phase III clinical studies for the treatment of Long-chain Fatty Acid Oxidation Disorders.
Product Name : Undisclosed
Product Type : Miscellaneous
Upfront Cash : Inapplicable
July 06, 2023
Lead Product(s) : Triheptanoin
Therapeutic Area : Genetic Disease
Highest Development Status : Phase III
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : Triheptanoin
Therapeutic Area : Genetic Disease
Study Phase : Approved FDF
Sponsor : Ultragenyx Pharmaceutical
Deal Size : Undisclosed
Deal Type : Partnership
Details : Orsini Pharmaceutical Services has been selected by Ultragenyx Pharmaceutical as a limited distribution specialty pharmacy partner for Dojolvi (triheptanoin). Dojolvi™ is indicated as a source of calories and fatty acids for the treatment of molecularl...
Product Name : Dojolvi
Product Type : Miscellaneous
Upfront Cash : Undisclosed
July 10, 2020
Lead Product(s) : Triheptanoin
Therapeutic Area : Genetic Disease
Highest Development Status : Approved FDF
Sponsor : Ultragenyx Pharmaceutical
Deal Size : Undisclosed
Deal Type : Partnership

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Lead Product(s) : Triheptanoin
Therapeutic Area : Undisclosed
Study Phase : Undisclosed
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable
Expanded Access to Triheptanoin
Details : Triheptanoin is a Other Small Molecule drug candidate, which is currently being evaluated in clinical studies for the treatment of undefined medical condition.
Product Name : Undisclosed
Product Type : Miscellaneous
Upfront Cash : Inapplicable
December 12, 2018
Lead Product(s) : Triheptanoin
Therapeutic Area : Undisclosed
Highest Development Status : Undisclosed
Sponsor : Undisclosed
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : Triheptanoin
Therapeutic Area : Genetic Disease
Study Phase : Phase I
Sponsor : Areeg El-Gharbawy
Deal Size : Inapplicable
Deal Type : Inapplicable
Anaplerotic Therapy Using Triheptanoin for Patients With Glycogen Storage Disease Type I
Details : Triheptanoin is a Other Small Molecule drug candidate, which is currently being evaluated in phase I clinical studies for the treatment of Glycogen Storage Disease Type I.
Product Name : Undisclosed
Product Type : Miscellaneous
Upfront Cash : Inapplicable
September 11, 2018
Lead Product(s) : Triheptanoin
Therapeutic Area : Genetic Disease
Highest Development Status : Phase I
Sponsor : Areeg El-Gharbawy
Deal Size : Inapplicable
Deal Type : Inapplicable

Lead Product(s) : Triheptanoin
Therapeutic Area : Genetic Disease
Study Phase : Phase II
Sponsor : Ultragenyx Pharmaceutical
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : Triheptanoin is a Other Small Molecule drug candidate, which is currently being evaluated in phase II clinical studies for the treatment of Glycogen Storage Disease Type VII.
Product Name : Undisclosed
Product Type : Miscellaneous
Upfront Cash : Inapplicable
August 22, 2018
Lead Product(s) : Triheptanoin
Therapeutic Area : Genetic Disease
Highest Development Status : Phase II
Sponsor : Ultragenyx Pharmaceutical
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : Triheptanoin
Therapeutic Area : Neurology
Study Phase : Phase I/ Phase II
Sponsor : Richard Bedlack, M.D., Ph.D.
Deal Size : Inapplicable
Deal Type : Inapplicable
A Pilot Trial of Triheptanoin for People With Amyotrophic Lateral Sclerosis (PALS)
Details : Triheptanoin is a Other Small Molecule drug candidate, which is currently being evaluated in phase I/ phase II clinical studies for the treatment of Amyotrophic Lateral Sclerosis.
Product Name : Undisclosed
Product Type : Miscellaneous
Upfront Cash : Inapplicable
April 24, 2018
Lead Product(s) : Triheptanoin
Therapeutic Area : Neurology
Highest Development Status : Phase I/ Phase II
Sponsor : Richard Bedlack, M.D., Ph.D.
Deal Size : Inapplicable
Deal Type : Inapplicable

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Lead Product(s) : Triheptanoin
Therapeutic Area : Genetic Disease
Study Phase : Undisclosed
Sponsor : Jerry Vockley
Deal Size : Inapplicable
Deal Type : Inapplicable
Treatment With UX007 for a Single Patient With GLUT1 Deficiency Syndrome
Details : Triheptanoin is a Other Small Molecule drug candidate, which is currently being evaluated in clinical studies for the treatment of Glut1 Deficiency Syndrome.
Product Name : Undisclosed
Product Type : Miscellaneous
Upfront Cash : Inapplicable
November 21, 2016
Lead Product(s) : Triheptanoin
Therapeutic Area : Genetic Disease
Highest Development Status : Undisclosed
Sponsor : Jerry Vockley
Deal Size : Inapplicable
Deal Type : Inapplicable
