[{"orgOrder":0,"company":"Chiesi Global Rare Diseases","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"Deferiprone","moa":"","graph1":"Genetic Disease","graph2":"Approved","graph3":"Chiesi Global Rare Diseases","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Chiesi Global Rare Diseases \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Chiesi Global Rare Diseases \/ Not Applicable"},{"orgOrder":0,"company":"Chiesi Global Rare Diseases","sponsor":"Protalix BioTherapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2023","type":"Not Applicable","leadProduct":"Pegunigalsidase alfa","moa":"","graph1":"Genetic Disease","graph2":"Approved","graph3":"Chiesi Global Rare Diseases","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Chiesi Global Rare Diseases \/ Chiesi Global Rare Diseases","highestDevelopmentStatusID":"12","companyTruncated":"Chiesi Global Rare Diseases \/ Chiesi Global Rare Diseases"},{"orgOrder":0,"company":"Chiesi Global Rare Diseases","sponsor":"Protalix BioTherapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2023","type":"Not Applicable","leadProduct":"Pegunigalsidase alfa-iwxj","moa":"","graph1":"Genetic Disease","graph2":"Approved","graph3":"Chiesi Global Rare Diseases","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Chiesi Global Rare Diseases \/ Chiesi Global Rare Diseases","highestDevelopmentStatusID":"12","companyTruncated":"Chiesi Global Rare Diseases \/ Chiesi Global Rare Diseases"},{"orgOrder":0,"company":"Chiesi Global Rare Diseases","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Peptide","year":"2024","type":"Not Applicable","leadProduct":"Metreleptin","moa":"","graph1":"Genetic Disease","graph2":"Approved","graph3":"Chiesi Global Rare Diseases","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Chiesi Global Rare Diseases \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Chiesi Global Rare Diseases \/ Not Applicable"},{"orgOrder":0,"company":"Chiesi Global Rare Diseases","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"Birch Triterpenes","moa":"","graph1":"Genetic Disease","graph2":"Approved","graph3":"Chiesi Global Rare Diseases","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Gel","sponsorNew":"Chiesi Global Rare Diseases \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Chiesi Global Rare Diseases \/ Not Applicable"},{"orgOrder":0,"company":"Chiesi Global Rare Diseases","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Pulmonary\/Respiratory Diseases","country":"","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"Tanimilast","moa":"","graph1":null,"graph2":null,"graph3":"Chiesi Global Rare Diseases","amount2":0,"highestDevelopmentShortName":null,"therapeuticAreaShortName":null,"amount2New":0,"dosageForm":"","sponsorNew":"Chiesi Global Rare Diseases \/ Not Applicable","highestDevelopmentStatusID":null,"companyTruncated":"Chiesi Global Rare Diseases \/ Not Applicable"}]

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                          Therapeutic Area by Lead Product

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                          Top Deals by Deal Size (USD bn)

                          01

                          American Pharma Summit
                          Not Confirmed
                          American Pharma Summit
                          Not Confirmed

                          Details : CHF6001 (tanimilast) is an inhaled phosphodiesterase-4 inhibitor, small molecule drug candidate which is currently being evaluated for the treatment of COPD and asthma.

                          Brand Name : CHF6001

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          May 16, 2024

                          Lead Product(s) : Tanimilast

                          Therapeutic Area : Pulmonary/Respiratory Diseases

                          Highest Development Status : Phase III

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          02

                          American Pharma Summit
                          Not Confirmed
                          American Pharma Summit
                          Not Confirmed

                          Details : Myalepta (metreleptin) is a USFDA approved leptin receptor activator, which is now approved by Health Canada for the treatment of congenital or acquired generalized lipodystrophy.

                          Brand Name : Myalepta

                          Molecule Type : Peptide

                          Upfront Cash : Not Applicable

                          February 05, 2024

                          Lead Product(s) : Metreleptin

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          03

                          American Pharma Summit
                          Not Confirmed
                          American Pharma Summit
                          Not Confirmed

                          Details : Filsuvez (birch triterpenes) is a topical gel which is approved for the treatment of wounds associated with dystrophic and junctional epidermolysis bullosa.

                          Brand Name : Filsuvez

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          December 19, 2023

                          Lead Product(s) : Birch Triterpenes

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          04

                          American Pharma Summit
                          Not Confirmed
                          American Pharma Summit
                          Not Confirmed

                          Details : Elfabrio (pegunigalsidase alfa-iwxj) is a PEGylated enzyme replacement therapy (ERT) for the treatment of adult patients with Fabry disease. It is a recombinant human α–Galactosidase–A enzyme expressed in plant-cell culture that is designed to provi...

                          Brand Name : Elfabrio

                          Molecule Type : Large molecule

                          Upfront Cash : Not Applicable

                          May 10, 2023

                          Lead Product(s) : Pegunigalsidase alfa-iwxj

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved

                          Recipient : Protalix BioTherapeutics

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          05

                          American Pharma Summit
                          Not Confirmed
                          American Pharma Summit
                          Not Confirmed

                          Details : PRX-102 (pegunigalsidase alfa), EC authorized, is a novel PEGylated, covalently crosslinked form of α-galactosidase-A developed as enzyme replacement therapy for fabry disease, was designed to increase plasma half-life and reduce immunogenicity, thereby...

                          Brand Name : PRX-102

                          Molecule Type : Large molecule

                          Upfront Cash : Not Applicable

                          May 05, 2023

                          Lead Product(s) : Pegunigalsidase alfa

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved

                          Recipient : Protalix BioTherapeutics

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          06

                          American Pharma Summit
                          Not Confirmed
                          American Pharma Summit
                          Not Confirmed

                          Details : Ferriprox™ (deferiprone) is a chelating agent with an affinity for ferric ion (iron III), being developed for the treatment of patients with transfusional iron overload due to thalassemia syndromes.

                          Brand Name : Ferriprox

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          March 31, 2023

                          Lead Product(s) : Deferiprone

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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