[{"orgOrder":0,"company":"Idorsia Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2021","type":"Not Applicable","leadProduct":"Lucerastat","moa":"GCS","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Idorsia Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Hard Gelatin Capsule","sponsorNew":"Idorsia Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Idorsia Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Idorsia Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2021","type":"Not Applicable","leadProduct":"Lucerastat","moa":"GCS","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Idorsia Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Hard Gelatin Capsule","sponsorNew":"Idorsia Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Idorsia Pharmaceuticals \/ Not Applicable"}]

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                          Top Deals by Deal Size (USD bn)

                          01

                          2024 ACI Convention
                          Not Confirmed
                          2024 ACI Convention
                          Not Confirmed

                          Details : Lucerastat demonstrated a substantial reduction in levels of the Fabry-disease biomarker plasma Gb3 after 6 months of treatment. The potential effect of lucerastat on kidney function over 6 months of treatment was evaluated at the interim analysis of the...

                          Brand Name : Undisclosed

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          December 13, 2021

                          Lead Product(s) : Lucerastat

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          02

                          2024 ACI Convention
                          Not Confirmed
                          2024 ACI Convention
                          Not Confirmed

                          Details : The safety, tolerability, pharmacodynamics, and pharmacokinetics of oral lucerastat were evaluated in study in adult patients with Fabry disease. In this single-center, open-label, randomized study, 10 patients with Fabry disease were randomized to lucer...

                          Brand Name : Undisclosed

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          October 11, 2021

                          Lead Product(s) : Lucerastat

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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