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    [{"orgOrder":0,"company":"Amgen Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2014","type":"Inapplicable","leadProduct":"Glycerol Phenylbutyrate","moa":"Glutamine","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Amgen Inc","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Liquid","sponsorNew":"Amgen Inc \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Amgen Inc \/ Undisclosed"},{"orgOrder":0,"company":"Amgen Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2017","type":"Inapplicable","leadProduct":"Glycerol Phenylbutyrate","moa":"Glutamine","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Amgen Inc","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Liquid","sponsorNew":"Amgen Inc \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Amgen Inc \/ Undisclosed"},{"orgOrder":0,"company":"National Jewish Health","sponsor":"University of Alabama at Birmingham | Children's Hospital of Philadelphia | Johns Hopkins University | Amgen Inc","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2014","type":"Inapplicable","leadProduct":"Glycerol Phenylbutyrate","moa":"Glutamine","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"National Jewish Health","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Liquid","sponsorNew":"National Jewish Health \/ University of Alabama at Birmingham | Children's Hospital of Philadelphia | Johns Hopkins University | Amgen Inc","highestDevelopmentStatusID":"7","companyTruncated":"National Jewish Health \/ University of Alabama at Birmingham | Children's Hospital of Philadelphia | Johns Hopkins University | Amgen Inc"},{"orgOrder":0,"company":"Amgen Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2010","type":"Inapplicable","leadProduct":"Glycerol Phenylbutyrate","moa":"Glutamine","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Amgen Inc","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Amgen Inc \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Amgen Inc \/ Undisclosed"},{"orgOrder":0,"company":"Amgen Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2011","type":"Inapplicable","leadProduct":"Glycerol Phenylbutyrate","moa":"Glutamine","graph1":"Genetic Disease","graph2":"Phase IV","graph3":"Amgen Inc","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Amgen Inc \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Amgen Inc \/ Undisclosed"},{"orgOrder":0,"company":"Amgen Inc","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Pharmacology\/Toxicology","country":"U.S.A","productType":"Miscellaneous","year":"2010","type":"Inapplicable","leadProduct":"Glycerol Phenylbutyrate","moa":"Glutamine","graph1":"Pharmacology\/Toxicology","graph2":"Phase I","graph3":"Amgen Inc","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Toxicology","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Amgen Inc \/ Undisclosed","highestDevelopmentStatusID":"6","companyTruncated":"Amgen Inc \/ Undisclosed"},{"orgOrder":0,"company":"The University of Pittsburgh School of Medicine","sponsor":"Amgen Inc","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2013","type":"Inapplicable","leadProduct":"Glycerol Phenylbutyrate","moa":"Glutamine","graph1":"Genetic Disease","graph2":"Phase I","graph3":"The University of Pittsburgh School of Medicine","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"The University of Pittsburgh School of Medicine \/ Amgen Inc","highestDevelopmentStatusID":"6","companyTruncated":"The University of Pittsburgh School of Medicine \/ Amgen Inc"},{"orgOrder":0,"company":"Weill Medical College of Cornell University","sponsor":"Children's Hospital Colorado | SLC6A1 Connect | STXBP1 Foundation | Clara Inspired | University of Pennsylvania Orphan Disease Center | Horizon Therapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Neurology","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Inapplicable","leadProduct":"Glycerol Phenylbutyrate","moa":"Glutamine","graph1":"Neurology","graph2":"Phase I","graph3":"Weill Medical College of Cornell University","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Neurology","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Weill Medical College of Cornell University \/ Children's Hospital Colorado | SLC6A1 Connect | STXBP1 Foundation | Clara Inspired | University of Pennsylvania Orphan Disease Center | Horizon Therapeutics","highestDevelopmentStatusID":"6","companyTruncated":"Weill Medical College of Cornell University \/ Children's Hospital Colorado | SLC6A1 Connect | STXBP1 Foundation | Clara Inspired | University of Pennsylvania Orphan Disease Center | Horizon Therapeutics"},{"orgOrder":0,"company":"Endo Pharm","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"IRELAND","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Glycerol Phenylbutyrate","moa":"Glutamine","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Endo Pharm","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Liquid","sponsorNew":"Endo Pharm \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Endo Pharm \/ Undisclosed"},{"orgOrder":0,"company":"Lupin Ltd","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"INDIA","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Glycerol Phenylbutyrate","moa":"Glutamine","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Lupin Ltd","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Liquid","sponsorNew":"Lupin Ltd \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Lupin Ltd \/ Undisclosed"}]

    Find Clinical Drug Pipeline Developments & Deals for Glycerol Phenylbutyrate

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    Glycerol Phenylbutyrate

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                            01

                            Lupin Ltd

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                            CPhI Milan
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                            Lead Product(s) : Glycerol Phenylbutyrate

                            Therapeutic Area : Genetic Disease

                            Study Phase : Approved FDF

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Lupin Launches Authorized Generic of RAVICTI in US

                            Details : Glycerol Phenylbutyrate, a small molecule product, targets glutamine to treat Urea Cycle Disorders (UCDs).

                            Product Name : Ravicti-Generic

                            Product Type : Miscellaneous

                            Upfront Cash : Inapplicable

                            October 24, 2025

                            Lead Product(s) : Glycerol Phenylbutyrate

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Approved FDF

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            02

                            Endo Pharm

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                            ISPOR Europe 2025
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                            Endo Pharm

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                            Lead Product(s) : Glycerol Phenylbutyrate

                            Therapeutic Area : Genetic Disease

                            Study Phase : Approved FDF

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Endo Launches First Generic RAVICTI® Oral Liquid in US

                            Details : Glycerol Phenylbutyrate, a small molecule product targeting glutamine, shows promise in treating urea cycle disorders (UCDs).

                            Product Name : Ravicti-Generic

                            Product Type : Miscellaneous

                            Upfront Cash : Inapplicable

                            October 20, 2025

                            Lead Product(s) : Glycerol Phenylbutyrate

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Approved FDF

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            03

                            Weill Medical College of Cornell University

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                            ISPOR Europe 2025
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                            Weill Medical College of Cornell University

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                            Not Confirmed
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                            Lead Product(s) : Glycerol Phenylbutyrate

                            Therapeutic Area : Neurology

                            Study Phase : Phase I

                            Sponsor : Children's Hospital Colorado | SLC6A1 Connect | STXBP1 Foundation | Clara Inspired | University of Pennsylvania Orphan Disease Center | Horizon Therapeutics

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Phenylbutyrate for Monogenetic Developmental and Epileptic Encephalopathy

                            Details : Glycerol Phenylbutyrate is a Other Small Molecule drug candidate, which is currently being evaluated in phase I clinical studies for the treatment of STXBP1 Encephalopathy-associated with Epilepsy.

                            Product Name : Undisclosed

                            Product Type : Miscellaneous

                            Upfront Cash : Inapplicable

                            June 23, 2021

                            Lead Product(s) : Glycerol Phenylbutyrate

                            Therapeutic Area : Neurology

                            Highest Development Status : Phase I

                            Sponsor : Children's Hospital Colorado | SLC6A1 Connect | STXBP1 Foundation | Clara Inspired | University of Pennsylvania Orphan Disease Center | Horizon Therapeutics

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            04

                            Amgen Inc

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                            ISPOR Europe 2025
                            Not Confirmed

                            Amgen Inc

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                            Lead Product(s) : Glycerol Phenylbutyrate

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase IV

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Study of Glycerol Phenylbutyrate & Sodium Phenylbutyrate in Phenylbutyrate Naïve Patients With Urea Cycle D...

                            Details : Ravicti is a Other Small Molecule drug candidate, which is currently being evaluated in phase IV clinical studies for the treatment of Urea Cycle Disorders, Inborn.

                            Product Name : Undisclosed

                            Product Type : Miscellaneous

                            Upfront Cash : Inapplicable

                            November 07, 2017

                            Lead Product(s) : Glycerol Phenylbutyrate

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase IV

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            05

                            National Jewish Health

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                            ISPOR Europe 2025
                            Not Confirmed

                            National Jewish Health

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                            Lead Product(s) : Glycerol Phenylbutyrate

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I/ Phase II

                            Sponsor : University of Alabama at Birmingham | Children's Hospital of Philadelphia | Johns Hopkins University | Amgen Inc

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Glycerol Phenylbutyrate Corrector Therapy For CF (Cystic Fibrosis)

                            Details : Ravicti is a Other Small Molecule drug candidate, which is currently being evaluated in phase I/ phase II clinical studies for the treatment of Cystic Fibrosis.

                            Product Name : Undisclosed

                            Product Type : Miscellaneous

                            Upfront Cash : Inapplicable

                            December 23, 2014

                            Lead Product(s) : Glycerol Phenylbutyrate

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : University of Alabama at Birmingham | Children's Hospital of Philadelphia | Johns Hopkins University | Amgen Inc

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            06

                            Amgen Inc

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                            ISPOR Europe 2025
                            Not Confirmed

                            Amgen Inc

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                            Lead Product(s) : Glycerol Phenylbutyrate

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase IV

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            A Study of the Safety, Efficacy and Pharmacokinetics of Glycerol Phenylbutyrate in Pediatric Subjects Under 2 ...

                            Details : Ravicti is a Other Small Molecule drug candidate, which is currently being evaluated in phase IV clinical studies for the treatment of Urea Cycle Disorders, Inborn.

                            Product Name : Undisclosed

                            Product Type : Miscellaneous

                            Upfront Cash : Inapplicable

                            September 22, 2014

                            Lead Product(s) : Glycerol Phenylbutyrate

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase IV

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            07

                            The University of Pittsburgh School of Medicine

                            U.S.A arrow
                            ISPOR Europe 2025
                            Not Confirmed

                            The University of Pittsburgh School of Medicine

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                            ISPOR Europe 2025
                            Not Confirmed
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                            Lead Product(s) : Glycerol Phenylbutyrate

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I

                            Sponsor : Amgen Inc

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Use of Ravictiâ„¢ in Patients With MCAD Deficiency With the 985A>G (K304E) Mutation

                            Details : Ravicti is a Other Small Molecule drug candidate, which is currently being evaluated in phase I clinical studies for the treatment of Medium Chain Acyl CoA Dehydrogenase Deficiency.

                            Product Name : Undisclosed

                            Product Type : Miscellaneous

                            Upfront Cash : Inapplicable

                            June 20, 2013

                            Lead Product(s) : Glycerol Phenylbutyrate

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I

                            Sponsor : Amgen Inc

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            08

                            Amgen Inc

                            U.S.A arrow
                            ISPOR Europe 2025
                            Not Confirmed

                            Amgen Inc

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                            ISPOR Europe 2025
                            Not Confirmed
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                            Lead Product(s) : Glycerol Phenylbutyrate

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase III

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Study of the Safety, Pharmacokinetics and Efficacy of HPN-100, in Pediatric Subjects With Urea Cycle Disorders...

                            Details : HPN-100 is a Other Small Molecule drug candidate, which is currently being evaluated in phase III clinical studies for the treatment of Urea Cycle Disorders, Inborn.

                            Product Name : Undisclosed

                            Product Type : Miscellaneous

                            Upfront Cash : Inapplicable

                            May 04, 2011

                            Lead Product(s) : Glycerol Phenylbutyrate

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase III

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            09

                            Amgen Inc

                            U.S.A arrow
                            ISPOR Europe 2025
                            Not Confirmed

                            Amgen Inc

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                            ISPOR Europe 2025
                            Not Confirmed
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                            Lead Product(s) : Glycerol Phenylbutyrate

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase IV

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            To Evaluate the Safety of Long-term Use of HPN-100 in the Management of Urea Cycle Disorders (UCDs)

                            Details : HPN-100 is a Other Small Molecule drug candidate, which is currently being evaluated in phase IV clinical studies for the treatment of Urea Cycle Disorders, Inborn.

                            Product Name : Undisclosed

                            Product Type : Miscellaneous

                            Upfront Cash : Inapplicable

                            December 10, 2010

                            Lead Product(s) : Glycerol Phenylbutyrate

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase IV

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            10

                            Amgen Inc

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                            ISPOR Europe 2025
                            Not Confirmed

                            Amgen Inc

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                            Not Confirmed
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                            Lead Product(s) : Glycerol Phenylbutyrate

                            Therapeutic Area : Pharmacology/Toxicology

                            Study Phase : Phase I

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Double-Blind Randomized Crossover Trial to Access Electrocardiogram Effects of HPN-100

                            Details : HPN-100 is a Other Small Molecule drug candidate, which is currently being evaluated in phase I clinical studies for the treatment of Drug-Related Side Effects and Adverse Reactions.

                            Product Name : Undisclosed

                            Product Type : Miscellaneous

                            Upfront Cash : Inapplicable

                            June 03, 2010

                            Lead Product(s) : Glycerol Phenylbutyrate

                            Therapeutic Area : Pharmacology/Toxicology

                            Highest Development Status : Phase I

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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