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Disorders","country":"U.S.A","productType":"Vitamins\/Minerals\/Inorganic Salts","year":"2016","type":"Inapplicable","leadProduct":"Coenzyme Q10","moa":"aldosterone antagonists (spironolactone type)","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"BPGbio","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Cream","sponsorNew":"BPGbio \/ Shasa Hu","highestDevelopmentStatusID":"6","companyTruncated":"BPGbio \/ Shasa Hu"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Michael Levy","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"JAPAN","productType":"Undisclosed","year":"2013","type":"Inapplicable","leadProduct":"C1 Esterase Inhibitor, Human","moa":"C1 esterase","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Takeda Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Takeda Pharmaceutical \/ Michael Levy","highestDevelopmentStatusID":"6","companyTruncated":"Takeda Pharmaceutical \/ Michael Levy"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Jirair Krikor Bedoyan","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"Triheptanoin","moa":"Triglyceride","graph1":"Rare Diseases and Disorders","graph2":"Phase I","graph3":"Ultragenyx Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Ultragenyx Pharmaceutical \/ Jirair Krikor Bedoyan","highestDevelopmentStatusID":"6","companyTruncated":"Ultragenyx Pharmaceutical \/ Jirair Krikor Bedoyan"}]

Find Drugs for Rare Diseases and Disorders in Phase I Clinical Development in UNITED STATES

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                          Top Deals by Deal Size (USD bn)

                          01

                          Lead Product(s) : SAR443809

                          Therapeutic Area : Rare Diseases and Disorders

                          Study Phase : Phase I

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Details : SAR443809 is a Antibody drug candidate, which is currently being evaluated in phase I clinical studies for the treatment of Hemoglobinuria, Paroxysmal.

                          Product Name : Undisclosed

                          Product Type : Antibody, Unconjugated

                          Upfront Cash : Inapplicable

                          March 22, 2024

                          Lead Product(s) : SAR443809

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase I

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Sanofi Company Banner

                          02

                          Lead Product(s) : GZ402666

                          Therapeutic Area : Rare Diseases and Disorders

                          Study Phase : Phase I

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Details : GZ402666 is a Enzyme drug candidate, which is currently being evaluated in phase I clinical studies for the treatment of Glycogen Storage Disease Type II.

                          Product Name : Undisclosed

                          Product Type : Enzyme

                          Upfront Cash : Inapplicable

                          July 12, 2013

                          Lead Product(s) : GZ402666

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase I

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Sanofi Company Banner

                          03

                          Lead Product(s) : Recombinant Human Acid Sphingomyelinase

                          Therapeutic Area : Rare Diseases and Disorders

                          Study Phase : Phase I

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Details : Recombinant Human Acid Sphingomyelinase is a Enzyme drug candidate, which is currently being evaluated in phase I clinical studies for the treatment of Niemann-Pick Disease, Type A.

                          Product Name : Undisclosed

                          Product Type : Enzyme

                          Upfront Cash : Inapplicable

                          November 07, 2012

                          Lead Product(s) : Recombinant Human Acid Sphingomyelinase

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase I

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Sanofi Company Banner

                          04

                          ISPE Annual Meeting
                          Not Confirmed
                          ISPE Annual Meeting
                          Not Confirmed

                          Details : Vorinostat is a Small Molecule drug candidate drug candidate, which is currently being evaluated in Phase I clinical studies for the treatment of Rett Syndrome.

                          Product Name : Undisclosed

                          Product Type : Miscellaneous

                          Upfront Cash : Inapplicable

                          September 02, 2025

                          Lead Product(s) : Vorinostat

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase I

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          05

                          ISPE Annual Meeting
                          Not Confirmed
                          ISPE Annual Meeting
                          Not Confirmed

                          Details : Vorinostat is a Small Molecule drug candidate drug candidate, which is currently being evaluated in Phase I clinical studies for the treatment of Pitt-Hopkins syndrome.

                          Product Name : Undisclosed

                          Product Type : Miscellaneous

                          Upfront Cash : Inapplicable

                          September 02, 2025

                          Lead Product(s) : Vorinostat

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase I

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          06

                          Jerry Vockley

                          Country arrow
                          ISPE Annual Meeting
                          Not Confirmed

                          Jerry Vockley

                          Country arrow
                          ISPE Annual Meeting
                          Not Confirmed

                          Details : Sodium Phenylbutyrate is a Other Small Molecule drug candidate, which is currently being evaluated in Phase I clinical studies for the treatment of 2-Hydroxyglutaricaciduria.

                          Product Name : Undisclosed

                          Product Type : Miscellaneous

                          Upfront Cash : Inapplicable

                          August 15, 2025

                          Lead Product(s) : Sodium Phenylbutyrate

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase I

                          Sponsor : Zevra Therapeutics

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          07

                          ISPE Annual Meeting
                          Not Confirmed
                          ISPE Annual Meeting
                          Not Confirmed

                          Details : KM-023 is a drug candidate, which is currently being evaluated in Phase I clinical studies for the treatment of Olmsted Syndrome.

                          Product Name : Undisclosed

                          Product Type : Undisclosed

                          Upfront Cash : Inapplicable

                          July 29, 2025

                          Lead Product(s) : KM-023

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase I

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          08

                          ISPE Annual Meeting
                          Not Confirmed
                          ISPE Annual Meeting
                          Not Confirmed

                          Details : RLF-TD011 is a differentiated hypotonic, acid-oxidizing solution formulated with hypochlorous acid, offering strong antimicrobial and anti-inflammatory properties for epidermolysis bullosa.

                          Product Name : Undisclosed

                          Product Type : Miscellaneous

                          Upfront Cash : Inapplicable

                          May 22, 2025

                          Lead Product(s) : Hypochlorous Acid

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase I

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          09

                          ISPE Annual Meeting
                          Not Confirmed
                          ISPE Annual Meeting
                          Not Confirmed

                          Details : Radiprodil is a Other Small Molecule drug candidate, which is currently being evaluated in Phase I clinical studies for the treatment of Tuberous Sclerosis.

                          Product Name : Undisclosed

                          Product Type : Miscellaneous

                          Upfront Cash : Inapplicable

                          May 16, 2025

                          Lead Product(s) : Radiprodil

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase I

                          Sponsor : Avance Clinical

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          10

                          ISPE Annual Meeting
                          Not Confirmed
                          ISPE Annual Meeting
                          Not Confirmed

                          Details : QRX003 is a topical lotion formulated with Invisicare and contains a broad-spectrum serine protease inhibitor, being developed for peeling skin syndrome.

                          Product Name : Undisclosed

                          Product Type : Miscellaneous

                          Upfront Cash : Inapplicable

                          May 14, 2025

                          Lead Product(s) : QRX003

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase I

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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