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    [{"orgOrder":0,"company":"Amgen Inc","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Antibody","year":"2023","type":"Inapplicable","leadProduct":"Inebilizumab","moa":"B-lymphocyte antigen CD19","graph1":"Rare Diseases and Disorders","graph2":"Phase IV","graph3":"Amgen Inc","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous","sponsorNew":"Amgen Inc \/ Inapplicable","highestDevelopmentStatusID":"11","companyTruncated":"Amgen Inc \/ Inapplicable"},{"orgOrder":0,"company":"Amgen Inc","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Antibody","year":"2022","type":"Inapplicable","leadProduct":"Inebilizumab","moa":"B-lymphocyte antigen CD19","graph1":"Rare Diseases and Disorders","graph2":"Phase IV","graph3":"Amgen Inc","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Intravenous","sponsorNew":"Amgen Inc \/ Inapplicable","highestDevelopmentStatusID":"11","companyTruncated":"Amgen Inc \/ Inapplicable"},{"orgOrder":0,"company":"Kyowa Kirin","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"JAPAN","productType":"Antibody","year":"2020","type":"Inapplicable","leadProduct":"KRN23","moa":"Fibroblast growth factor 23","graph1":"Rare Diseases and Disorders","graph2":"Phase IV","graph3":"Kyowa Kirin","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Kyowa Kirin \/ Inapplicable","highestDevelopmentStatusID":"11","companyTruncated":"Kyowa Kirin \/ Inapplicable"},{"orgOrder":0,"company":"Regeneron Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Antibody","year":"2023","type":"Inapplicable","leadProduct":"Pozelimab","moa":"Complement C5","graph1":"Rare Diseases and Disorders","graph2":"Phase IV","graph3":"Regeneron Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Regeneron Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"11","companyTruncated":"Regeneron Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Alexion Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Antibody","year":"2020","type":"Inapplicable","leadProduct":"Eculizumab","moa":"Complement C5","graph1":"Rare Diseases and Disorders","graph2":"Phase IV","graph3":"Alexion Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Alexion Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"11","companyTruncated":"Alexion Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Kyowa Kirin","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"JAPAN","productType":"Antibody","year":"2021","type":"Inapplicable","leadProduct":"KRN23","moa":"Fibroblast growth factor 23","graph1":"Rare Diseases and Disorders","graph2":"Phase IV","graph3":"Kyowa Kirin","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Solution","sponsorNew":"Kyowa Kirin \/ Inapplicable","highestDevelopmentStatusID":"11","companyTruncated":"Kyowa Kirin \/ Inapplicable"},{"orgOrder":0,"company":"Kyowa Kirin","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"JAPAN","productType":"Antibody","year":"2021","type":"Inapplicable","leadProduct":"KRN23","moa":"Fibroblast growth factor 23","graph1":"Rare Diseases and Disorders","graph2":"Phase IV","graph3":"Kyowa Kirin","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Solution","sponsorNew":"Kyowa Kirin \/ Inapplicable","highestDevelopmentStatusID":"11","companyTruncated":"Kyowa Kirin \/ Inapplicable"},{"orgOrder":0,"company":"Hansoh Pharma","sponsor":"Amgen Inc","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"CHINA","productType":"Antibody","year":"2024","type":"Inapplicable","leadProduct":"Inebilizumab","moa":"B-lymphocyte antigen CD19","graph1":"Rare Diseases and Disorders","graph2":"Phase IV","graph3":"Hansoh Pharma","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Hansoh Pharma \/ Horizon Therapeutics","highestDevelopmentStatusID":"11","companyTruncated":"Hansoh Pharma \/ Horizon Therapeutics"},{"orgOrder":0,"company":"Neurotech Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2025","type":"Inapplicable","leadProduct":"NT-501","moa":"atropine derivatives","graph1":"Rare Diseases and Disorders","graph2":"Phase IV","graph3":"Neurotech Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Implant","sponsorNew":"Neurotech Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"11","companyTruncated":"Neurotech Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Alexion Pharmaceuticals","sponsor":"AstraZeneca","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Antibody","year":"2023","type":"Inapplicable","leadProduct":"Eculizumab","moa":"Complement C5","graph1":"Rare Diseases and Disorders","graph2":"Phase IV","graph3":"Alexion Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Alexion Pharmaceuticals \/ AstraZeneca","highestDevelopmentStatusID":"11","companyTruncated":"Alexion Pharmaceuticals \/ AstraZeneca"},{"orgOrder":0,"company":"Neurotech Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"NT-501","moa":"atropine derivatives","graph1":"Rare Diseases and Disorders","graph2":"Phase IV","graph3":"Neurotech Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Implant","sponsorNew":"Neurotech Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"11","companyTruncated":"Neurotech Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Neurotech Pharmaceuticals","sponsor":"The Lowy Medical Research Institute Limited","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Cell and Gene therapy","year":"2021","type":"Inapplicable","leadProduct":"NT-501","moa":"atropine derivatives","graph1":"Rare Diseases and Disorders","graph2":"Phase IV","graph3":"Neurotech Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Implant","sponsorNew":"Neurotech Pharmaceuticals \/ The Lowy Medical Research Institute Limited","highestDevelopmentStatusID":"11","companyTruncated":"Neurotech Pharmaceuticals \/ The Lowy Medical Research Institute Limited"},{"orgOrder":0,"company":"Regeneron Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Antibody","year":"2025","type":"Inapplicable","leadProduct":"Pozelimab","moa":"Complement C5","graph1":"Rare Diseases and Disorders","graph2":"Phase IV","graph3":"Regeneron Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Regeneron Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Regeneron Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Chinese SLE Treatment And Research Group","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Other Small Molecule","year":"2025","type":"Inapplicable","leadProduct":"Deucravacitinib","moa":"Tyrosine-protein kinase TYK2","graph1":"Rare Diseases and Disorders","graph2":"Phase IV","graph3":"Chinese SLE Treatment And Research Group","amount2":0,"highestDevelopmentShortName":"Ph IV","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Chinese SLE Treatment And Research Group \/ Undisclosed","highestDevelopmentStatusID":"11","companyTruncated":"Chinese SLE Treatment And Research Group \/ Undisclosed"}]

    Find Clinical Drug Development Pipelines & Deals | PipelineProspector

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                            01

                            Regeneron Pharmaceuticals

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                            Regeneron Pharmaceuticals

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                            Lead Product(s) : Pozelimab

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase IV

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Pozelimab Safety In Pediatric CHAPLE Disease (1–5 Years)

                            Details : Pozelimab is a Antibody drug candidate, which is currently being evaluated in Phase IV clinical studies for the treatment of Chaple Disease.

                            Product Name : Undisclosed

                            Product Type : Antibody

                            Upfront Cash : Inapplicable

                            August 26, 2025

                            Lead Product(s) : Pozelimab

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase IV

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            02

                            Chinese SLE Treatment And Research Group

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                            BIOHK2025
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                            Chinese SLE Treatment And Research Group

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                            BIOHK2025
                            Not Confirmed
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                            Lead Product(s) : Deucravacitinib

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase IV

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            The Efficacy and Safety of Deucravacitinib in Takayasu's Arteritis

                            Details : Deucravacitinib is a Other Small Molecule drug candidate, which is currently being evaluated in Phase IV clinical studies for the treatment of Takayasu Arteritis.

                            Product Name : Undisclosed

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            June 10, 2025

                            Lead Product(s) : Deucravacitinib

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase IV

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            03

                            Neurotech Pharmaceuticals

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                            BIOHK2025
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                            Neurotech Pharmaceuticals

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                            BIOHK2025
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                            Lead Product(s) : NT-501

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase IV

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Phase 4 Study: Long-term Safety and Efficacy of NT-501 in MacTel Type 2, Including Sham Procedure Participants

                            Details : Undisclosed

                            Product Name : Undisclosed

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Inapplicable

                            May 14, 2025

                            Lead Product(s) : NT-501

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase IV

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            04

                            Hansoh Pharma

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                            BIOHK2025
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                            Hansoh Pharma

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                            BIOHK2025
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                            Lead Product(s) : Inebilizumab

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase IV

                            Sponsor : Amgen Inc

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            A Clinical Research Study of Inebilizumab in Neuromyelitis Optica Spectrum Disorders

                            Details : Undisclosed

                            Product Name : Undisclosed

                            Product Type : Antibody

                            Upfront Cash : Inapplicable

                            January 18, 2024

                            Lead Product(s) : Inebilizumab

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase IV

                            Sponsor : Amgen Inc

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            05

                            Amgen Inc

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                            BIOHK2025
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                            Amgen Inc

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                            BIOHK2025
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                            Lead Product(s) : Inebilizumab

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase IV

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Long-term, Open-label, Safety Study of Inebilizumab in Neuromyelitis Optica Spectrum Disorder (NMOSD)

                            Details : Undisclosed

                            Product Name : Undisclosed

                            Product Type : Antibody

                            Upfront Cash : Inapplicable

                            December 22, 2023

                            Lead Product(s) : Inebilizumab

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase IV

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            06

                            F. Hoffmann-La Roche

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                            BIOHK2025
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                            F. Hoffmann-La Roche

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                            BIOHK2025
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                            Lead Product(s) : Satralizumab

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase IV

                            Sponsor : Chugai Pharmaceutical

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            A Study In Neuromyelitis Optica Spectrum Disorder (NMOSD) With Satralizumab As An Intervention

                            Details : Undisclosed

                            Product Name : Undisclosed

                            Product Type : Antibody

                            Upfront Cash : Inapplicable

                            March 08, 2022

                            Lead Product(s) : Satralizumab

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase IV

                            Sponsor : Chugai Pharmaceutical

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            07

                            Jazz Pharmaceuticals

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                            BIOHK2025
                            Not Confirmed

                            Jazz Pharmaceuticals

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                            BIOHK2025
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                            Lead Product(s) : Cannabidiol

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase IV

                            Sponsor : Jazz Pharmaceuticals

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Assessment of Potential for Chronic Liver Injury in Participants Treated With Epidiolex (Cannabidiol) Oral Sol...

                            Details : Undisclosed

                            Product Name : Undisclosed

                            Product Type : Controlled Substance

                            Upfront Cash : Inapplicable

                            September 16, 2021

                            Lead Product(s) : Cannabidiol

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase IV

                            Sponsor : Jazz Pharmaceuticals

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            08

                            Apellis Pharmaceuticals

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                            BIOHK2025
                            Not Confirmed

                            Apellis Pharmaceuticals

                            U.S.A arrow
                            BIOHK2025
                            Not Confirmed
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                            Lead Product(s) : Pegcetacoplan

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase IV

                            Sponsor : SFJ Pharmaceuticals

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Apellis to Present Long-Term Pegcetacoplan Data in PNH at the European Hematology Association Virtual Congress

                            Details : In the study, EMPAVELI met the primary endpoint, demonstrating superiority to Soliris for the change from baseline in hemoglobin level at Week 16 with an adjusted mean increase of 3.84 g/dL of hemoglobin.

                            Product Name : Empaveli

                            Product Type : Peptide

                            Upfront Cash : Inapplicable

                            May 14, 2021

                            Lead Product(s) : Pegcetacoplan

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase IV

                            Sponsor : SFJ Pharmaceuticals

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            09

                            Kyowa Kirin

                            Japan arrow
                            BIOHK2025
                            Not Confirmed

                            Kyowa Kirin

                            Japan arrow
                            BIOHK2025
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                            Lead Product(s) : KRN23

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase IV

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Study to Assess the Safety, Pharmacokinetics and Efficacy of KRN23 in Adult Chinese Patients With XLH

                            Details : Undisclosed

                            Product Name : Undisclosed

                            Product Type : Antibody

                            Upfront Cash : Inapplicable

                            April 12, 2021

                            Lead Product(s) : KRN23

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase IV

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            10

                            Kyowa Kirin

                            Japan arrow
                            BIOHK2025
                            Not Confirmed

                            Kyowa Kirin

                            Japan arrow
                            BIOHK2025
                            Not Confirmed
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                            Lead Product(s) : KRN23

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase IV

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Study to Assess the Safety, Pharmacokinetics and Efficacy of KRN23 in Pediatric Chinese Patients With XLH

                            Details : Undisclosed

                            Product Name : Undisclosed

                            Product Type : Antibody

                            Upfront Cash : Inapplicable

                            April 12, 2021

                            Lead Product(s) : KRN23

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase IV

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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