[{"orgOrder":0,"company":"Pharming","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Infections and Infectious Diseases","country":"NETHERLANDS","productType":"Large molecule","year":"2020","type":"Not Applicable","leadProduct":"Conestat alfa","moa":"C1 esterase","graph1":"Infections and Infectious Diseases","graph2":"Undisclosed","graph3":"Pharming","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Infectious Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Pharming \/ Not Applicable","highestDevelopmentStatusID":"1","companyTruncated":"Pharming \/ Not Applicable"},{"orgOrder":0,"company":"Pharming","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Infections and Infectious Diseases","country":"","productType":"Large molecule","year":"2020","type":"Not Applicable","leadProduct":"Conestat alfa","moa":"C1 esterase","graph1":"Infections and Infectious Diseases","graph2":"Phase II","graph3":"Pharming","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Infectious Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Pharming \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Pharming \/ Not Applicable"},{"orgOrder":0,"company":"Pharming","sponsor":"Novartis Pharmaceuticals Corporation","pharmaFlowCategory":"DU","therapeuticArea":"Immunology","country":"","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Leniolisib","moa":"PI3K delta","graph1":"Immunology","graph2":"Phase II\/ Phase III","graph3":"Pharming","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Immunology","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Pharming \/ Novartis","highestDevelopmentStatusID":"9","companyTruncated":"Pharming \/ Novartis"},{"orgOrder":0,"company":"Pharming","sponsor":"Orchard Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2021","type":"Collaboration","leadProduct":"Autologous Hematopoietic Stem Cell Gene Therapy","moa":"C1 esterase","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Pharming","amount2":0.20999999999999999,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.20999999999999999,"dosageForm":"","sponsorNew":"Pharming \/ Pharming Group","highestDevelopmentStatusID":"4","companyTruncated":"Pharming \/ Pharming Group"},{"orgOrder":0,"company":"Pharming","sponsor":"NewBridge Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2021","type":"Agreement","leadProduct":"Conestat Alfa","moa":"C1 esterase","graph1":"Genetic Disease","graph2":"Approved","graph3":"Pharming","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Powder for Solution for Injection","sponsorNew":"Pharming \/ NewBridge Pharmaceuticals","highestDevelopmentStatusID":"12","companyTruncated":"Pharming \/ NewBridge Pharmaceuticals"},{"orgOrder":0,"company":"Pharming","sponsor":"Novartis Pharmaceuticals Corporation","pharmaFlowCategory":"DU","therapeuticArea":"Immunology","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Leniolisib","moa":"PI3K delta","graph1":"Immunology","graph2":"Phase II\/ Phase III","graph3":"Pharming","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Immunology","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Pharming \/ Novartis","highestDevelopmentStatusID":"9","companyTruncated":"Pharming \/ Novartis"},{"orgOrder":0,"company":"Pharming","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Immunology","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Leniolisib","moa":"PI3K delta","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Pharming","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Pharming \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Pharming \/ Not Applicable"},{"orgOrder":0,"company":"Pharming","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Leniolisib","moa":"PI3K delta","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Pharming","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Pharming \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Pharming \/ Not Applicable"},{"orgOrder":0,"company":"Pharming","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Immunology","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Leniolisib","moa":"PI3K delta","graph1":"Immunology","graph2":"Phase III","graph3":"Pharming","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Immunology","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Pharming \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Pharming \/ Not Applicable"},{"orgOrder":0,"company":"Pharming","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Leniolisib","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Pharming","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Pharming \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Pharming \/ Not Applicable"},{"orgOrder":0,"company":"Pharming","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Leniolisib","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Pharming","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Pharming \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Pharming \/ Not Applicable"},{"orgOrder":0,"company":"Pharming","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Leniolisib","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Pharming","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Pharming \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Pharming \/ Not Applicable"},{"orgOrder":0,"company":"Pharming","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"Leniolisib","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Pharming","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Pharming \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Pharming \/ Not 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Disorders","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"Leniolisib","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Pharming","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Pharming \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Pharming \/ Not Applicable"},{"orgOrder":0,"company":"Pharming","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"Leniolisib","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Approved","graph3":"Pharming","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"Pharming \/ Not 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                          Therapeutic Area by Lead Product

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                          Top Deals by Deal Size (USD bn)

                          01

                          Medlab Asia & Asia Health
                          Not Confirmed
                          Medlab Asia & Asia Health
                          Not Confirmed

                          Details : Joenja (leniolisib) is an oral small molecule PI3K delta inhibitor, which is currently being evaluated for the treatment of primary immunodeficiency disorders.

                          Brand Name : Joenja

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          October 10, 2024

                          Lead Product(s) : Leniolisib

                          Therapeutic Area : Immunology

                          Highest Development Status : Phase II

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          02

                          Medlab Asia & Asia Health
                          Not Confirmed
                          Medlab Asia & Asia Health
                          Not Confirmed

                          Details : Joenja (leniolisib) is an oral PI3K delta inhibitor, the first targeted treatment for activated PI3K delta syndrome in patients aged 12 and older, now being developed for children aged 4 to 11.

                          Brand Name : Joenja

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          April 08, 2024

                          Lead Product(s) : Leniolisib

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase III

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          03

                          Medlab Asia & Asia Health
                          Not Confirmed
                          Medlab Asia & Asia Health
                          Not Confirmed

                          Details : Joenja (leniolisib) is an oral small molecule PI3Kẟ inhibitor approved in US as the first and only targeted treatment of activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) in adult and pediatric patients 12 years of age and older.

                          Brand Name : Joenja

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          December 13, 2023

                          Lead Product(s) : Leniolisib

                          Therapeutic Area : Immunology

                          Highest Development Status : Phase II

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          04

                          Medlab Asia & Asia Health
                          Not Confirmed
                          Medlab Asia & Asia Health
                          Not Confirmed

                          Details : Leniolisib is an oral small molecule phosphoinositide 3-kinase delta inhibitor approved in the US as the first and only targeted treatment of activated phosphoinositide 3-kinase delta syndrome in adult and pediatric patients 12 years of age and older.

                          Brand Name : CDZ173

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          November 21, 2023

                          Lead Product(s) : Leniolisib

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase III

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          05

                          Medlab Asia & Asia Health
                          Not Confirmed
                          Medlab Asia & Asia Health
                          Not Confirmed

                          Details : Joenja (leniolisib) is an oral, selective PI3K-delta inhibitor which inhibits the signalling pathways that lead to increased production of PIP3, hyperactivity of the downstream mTOR/AKT pathway, and to the dysregulation of B and T cells in patients with ...

                          Brand Name : Joenja

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          November 10, 2023

                          Lead Product(s) : Leniolisib

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase III

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          06

                          Medlab Asia & Asia Health
                          Not Confirmed
                          Medlab Asia & Asia Health
                          Not Confirmed

                          Details : Joenja (leniolisib) is an oral, selective PI3K-delta inhibitor which inhibits the signalling pathways that lead to increased production of PIP3, hyperactivity of the downstream mTOR/AKT pathway, and to the dysregulation of B and T cells in patients with ...

                          Brand Name : Joenja

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          August 09, 2023

                          Lead Product(s) : Leniolisib

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase III

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          07

                          Medlab Asia & Asia Health
                          Not Confirmed
                          Medlab Asia & Asia Health
                          Not Confirmed

                          Details : Under the agreement, Pharming will sell its Rare Pediatric Disease Priority Review Voucher, in connection with the approval of Joenja® (leniolisib), an oral, selective PI3Kδ inhibitor, for activated PI3Kδ syndrome, a rare and progressive primary immun...

                          Brand Name : Joenja

                          Molecule Type : Small molecule

                          Upfront Cash : Undisclosed

                          June 01, 2023

                          Lead Product(s) : Leniolisib

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Approved

                          Sponsor : Novartis Pharmaceuticals Corporation

                          Deal Size : $21.1 million

                          Deal Type : Divestment

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                          08

                          Medlab Asia & Asia Health
                          Not Confirmed
                          Medlab Asia & Asia Health
                          Not Confirmed

                          Details : Joenja (leniolisib) is an oral, selective PI3K-delta inhibitor which inhibits the signalling pathways that lead to increased production of PIP3, hyperactivity of the downstream mTOR/AKT pathway, and to the dysregulation of B and T cells in patients with ...

                          Brand Name : Joenja

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          March 24, 2023

                          Lead Product(s) : Leniolisib

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          09

                          Medlab Asia & Asia Health
                          Not Confirmed
                          Medlab Asia & Asia Health
                          Not Confirmed

                          Details : Leniolisib, an oral, selective phosphoinositide 3-kinase delta (PI3Kδ) inhibitor. It is being investigated in children with activated phosphoinositide 3-kinase delta syndrome (APDS).

                          Brand Name : Undisclosed

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          February 21, 2023

                          Lead Product(s) : Leniolisib

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          10

                          Medlab Asia & Asia Health
                          Not Confirmed
                          Medlab Asia & Asia Health
                          Not Confirmed

                          Details : Leniolisib is a small-molecule inhibitor of the delta isoform of the 110 kDa catalytic subunit of class IA PI3K. PI3K delta is a valid and potentially effective therapeutic target for immune diseases such as APDS.

                          Brand Name : CDZ173

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          February 16, 2023

                          Lead Product(s) : Leniolisib

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase III

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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