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                          01

                          2024 ACI Convention
                          Not Confirmed
                          2024 ACI Convention
                          Not Confirmed

                          Details : Orkambi is a combination of lumacaftor which improves the conformational stability of F508del-CFTR and ivacaftor, a CFTR potentiator. It is being used for cystic fibrosis in patients aged 1 year and older who have two copies of the F508del mutation in CF...

                          Brand Name : Orkambi

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          July 05, 2023

                          Lead Product(s) : Lumacaftor,Ivacaftor

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved

                          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 : Orkambi is a combination of lumacaftor which improves the conformational stability of F508del-CFTR and ivacaftor is a CFTR potentiator. It is being used for cystic fibrosis in patients aged 1 year and older who have two copies of the F508del mutation in ...

                          Brand Name : Orkambi

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          April 27, 2023

                          Lead Product(s) : Lumacaftor,Ivacaftor

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          03

                          2024 ACI Convention
                          Not Confirmed
                          2024 ACI Convention
                          Not Confirmed

                          Details : ORKAMBI® (lumacaftor/ivacaftor) is an oral medicine that is a combination of lumacaftor and ivacaftor. Lumacaftor is designed to increase the amount of mature protein at the cell surface by targeting the processing and trafficking defect of the F508del-...

                          Brand Name : Orkambi

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          September 02, 2022

                          Lead Product(s) : Lumacaftor,Ivacaftor

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          04

                          2024 ACI Convention
                          Not Confirmed
                          2024 ACI Convention
                          Not Confirmed

                          Details : Agreement enables the possibility of rapid patient access to future triple combination regimen (elexacaftor/tezacaftor/ivacaftor and ivacaftor) once approved in Switzerland .

                          Brand Name : Undisclosed

                          Molecule Type : Small molecule

                          Upfront Cash : Undisclosed

                          April 21, 2020

                          Lead Product(s) : Lumacaftor,Ivacaftor

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved

                          Sponsor : FSIO

                          Deal Size : Undisclosed

                          Deal Type : Agreement

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