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Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2022","type":"Inapplicable","leadProduct":"Elexacaftor","moa":"||Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"Elexacaftor","moa":"||Cystic fibrosis transmembrane conductance 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Undisclosed"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Spanish Government","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2021","type":"Agreement","leadProduct":"Ivacaftor","moa":"||Cystic fibrosis transmembrane conductance regulator","graph1":"Genetic Disease","graph2":"Approved FDF","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved FDF","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"Vertex Pharmaceuticals \/ Spanish Government","highestDevelopmentStatusID":"15","companyTruncated":"Vertex Pharmaceuticals \/ Spanish Government"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"Ivacaftor","moa":"||Cystic fibrosis 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                          01

                          FNCE
                          Not Confirmed
                          FNCE
                          Not Confirmed

                          Details : Kaftrio (ivacaftor/tezacaftor/elexacaftor) in combination with ivacaftor is an oral medicine designed to increase the quantity and function of the CFTR protein at the cell surface.

                          Product Name : Kaftrio

                          Product Type : Miscellaneous

                          Upfront Cash : Inapplicable

                          April 07, 2025

                          Lead Product(s) : Ivacaftor,Elexacaftor,Tezacaftor

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved FDF

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          02

                          FNCE
                          Not Confirmed
                          FNCE
                          Not Confirmed

                          Details : Kaftrio (ivacaftor/tezacaftor/elexacaftor) is an oral medicine designed to bind on CFTR protein, which is investigated for cystic fibrosis in patients aged 2 years & above.

                          Product Name : Kaftrio

                          Product Type : Miscellaneous

                          Upfront Cash : Inapplicable

                          February 28, 2025

                          Lead Product(s) : Ivacaftor,Elexacaftor,Tezacaftor

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          03

                          FNCE
                          Not Confirmed
                          FNCE
                          Not Confirmed

                          Details : Trikafta (ivacaftor/tezacaftor/elexacaftor) is an oral drug designed to binding to different sites on the CFTR protein, indicated for F508del-mutated cystic fibrosis in patients aged 2 years & above.

                          Product Name : Trikafta

                          Product Type : Miscellaneous

                          Upfront Cash : Inapplicable

                          December 20, 2024

                          Lead Product(s) : Elexacaftor,Ivacaftor,Tezacaftor

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved FDF

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          04

                          FNCE
                          Not Confirmed
                          FNCE
                          Not Confirmed

                          Details : Elexacaftor is a Other Small Molecule drug candidate, which is currently being evaluated in phase III clinical studies for the treatment of Cystic Fibrosis.

                          Product Name : Undisclosed

                          Product Type : Miscellaneous

                          Upfront Cash : Inapplicable

                          June 14, 2024

                          Lead Product(s) : Elexacaftor

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          05

                          FNCE
                          Not Confirmed
                          FNCE
                          Not Confirmed

                          Details : Kaftrio (ivacaftor/tezacaftor/elexacaftor) is an oral medicine designed to binding to different sites on the CFTR protein, which is investigated for the treatment of cystic fibrosis in patients aged 2 years & above.

                          Product Name : Kaftrio

                          Product Type : Miscellaneous

                          Upfront Cash : Inapplicable

                          November 24, 2023

                          Lead Product(s) : Ivacaftor,Elexacaftor,Tezacaftor

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved FDF

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          06

                          FNCE
                          Not Confirmed
                          FNCE
                          Not Confirmed

                          Details : Kaftrio (ivacaftor/tezacaftor/elexacaftor) is an oral medicine designed to binding to different sites on the CFTR protein, which is investigated for the treatment of cystic fibrosis in patients aged 2 years & above.

                          Product Name : Kaftrio

                          Product Type : Miscellaneous

                          Upfront Cash : Inapplicable

                          November 23, 2023

                          Lead Product(s) : Ivacaftor,Elexacaftor,Tezacaftor

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved FDF

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          07

                          FNCE
                          Not Confirmed
                          FNCE
                          Not Confirmed

                          Details : Kaftrio (ivacaftor/tezacaftor/elexacaftor) is an oral medicine designed to binding to different sites on the CFTR protein, which is investigated for the treatment of children With cystic fibrosis ages 2 through 5.

                          Product Name : Trikafta

                          Product Type : Miscellaneous

                          Upfront Cash : Inapplicable

                          September 15, 2023

                          Lead Product(s) : Elexacaftor,Ivacaftor,Tezacaftor

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved FDF

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          08

                          FNCE
                          Not Confirmed
                          FNCE
                          Not Confirmed

                          Details : Elexacaftor is a Other Small Molecule drug candidate, which is currently being evaluated in phase III clinical studies for the treatment of Cystic Fibrosis.

                          Product Name : Undisclosed

                          Product Type : Miscellaneous

                          Upfront Cash : Inapplicable

                          May 31, 2023

                          Lead Product(s) : Elexacaftor

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          09

                          FNCE
                          Not Confirmed
                          FNCE
                          Not Confirmed

                          Details : Trikafta is a combination drug where elexacaftor and tezacaftor bind to different sites of CFTR and additionally facilitates the cellular processing and trafficking of mutated CFTR. Ivacaftor potentiates the channel open probability of the CFTR protein a...

                          Product Name : Trikafta

                          Product Type : Miscellaneous

                          Upfront Cash : Inapplicable

                          April 26, 2023

                          Lead Product(s) : Elexacaftor,Ivacaftor,Tezacaftor

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved FDF

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          10

                          FNCE
                          Not Confirmed
                          FNCE
                          Not Confirmed

                          Details : TRIKAFTA (elexacaftor/tezacaftor/ivacaftor and ivacaftor), is prescription medicine for treatment of cystic fibrosis. Based on analysis through Week 144 of 192-week study, people receiving TRIKAFTA maintained improvement in lung function, respiratory sym...

                          Product Name : Trikafta

                          Product Type : Miscellaneous

                          Upfront Cash : Inapplicable

                          November 03, 2022

                          Lead Product(s) : Elexacaftor,Ivacaftor,Tezacaftor

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved FDF

                          Sponsor : Undisclosed

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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