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Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Vertex Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Elexacaftor","moa":"CFTR","graph1":"Genetic Disease","graph2":"Approved","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Vertex Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Ivacaftor","moa":"CFTR","graph1":"Genetic Disease","graph2":"Approved","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Vertex Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Ivacaftor","moa":"CFTR","graph1":"Genetic Disease","graph2":"Approved","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Vertex Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2021","type":"Not Applicable","leadProduct":"Ivacaftor","moa":"CFTR","graph1":"Genetic Disease","graph2":"Approved","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Vertex Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2021","type":"Not Applicable","leadProduct":"Ivacaftor","moa":"CFTR","graph1":"Genetic Disease","graph2":"Approved","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Film-coated tablet","sponsorNew":"Vertex Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Vertex Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2021","type":"Not Applicable","leadProduct":"Ivacaftor","moa":"CFTR","graph1":"Genetic Disease","graph2":"Approved","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Film-coated tablet","sponsorNew":"Vertex Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Vertex Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2021","type":"Not Applicable","leadProduct":"Elexacaftor","moa":"CFTR","graph1":"Genetic Disease","graph2":"Approved","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Vertex Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"AIFA","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2021","type":"Agreement","leadProduct":"Ivacaftor","moa":"CFTR","graph1":"Genetic Disease","graph2":"Approved","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Film-coated tablet","sponsorNew":"Vertex Pharmaceuticals \/ AIFA","highestDevelopmentStatusID":"12","companyTruncated":"Vertex Pharmaceuticals \/ AIFA"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"French Health Authorities for the cystic fibrosis","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2021","type":"Agreement","leadProduct":"Ivacaftor","moa":"CFTR","graph1":"Genetic Disease","graph2":"Approved","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Film-coated tablet","sponsorNew":"Vertex Pharmaceuticals \/ French Health Authorities for the cystic fibrosis","highestDevelopmentStatusID":"12","companyTruncated":"Vertex Pharmaceuticals \/ French Health Authorities for the cystic fibrosis"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2021","type":"Not Applicable","leadProduct":"Deutivacaftor","moa":"CFTR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Vertex Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2021","type":"Not 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Applicable","highestDevelopmentStatusID":"12","companyTruncated":"Vertex Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Spanish government","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2021","type":"Agreement","leadProduct":"Ivacaftor","moa":"CFTR","graph1":"Genetic Disease","graph2":"Approved","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Approved","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Film-Coated Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Spanish government","highestDevelopmentStatusID":"12","companyTruncated":"Vertex Pharmaceuticals \/ Spanish government"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2022","type":"Not 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Find Clinical Drug Pipeline Developments & Deals for Tezacaftor

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                          01

                          2024 ACI Convention
                          Not Confirmed
                          2024 ACI Convention
                          Not Confirmed

                          Details : VX-121/VX-661/VX-561 (vanzacaftor/tezacaftor/deutivacaftor) is a combination of three CFTR modulators. It is being evaluated in phase 3 clinial trials for the treatment of Cystic Fibrosis.

                          Brand Name : VX-121

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          July 02, 2024

                          Lead Product(s) : Vanzacaftor,Tezacaftor,Deutivacaftor

                          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 : VX-121/VX-661/VX-561 (vanzacaftor/tezacaftor/deutivacaftor) is a combination of three CFTR modulators. It is being evaluated in phase 3 clinial trials for the treatment of Cystic Fibrosis.

                          Brand Name : VX-121

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          February 05, 2024

                          Lead Product(s) : Vanzacaftor,Tezacaftor,Deutivacaftor

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          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 : 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.

                          Brand Name : Kaftrio

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          November 24, 2023

                          Lead Product(s) : Ivacaftor,Tezacaftor,Elexacaftor

                          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 : 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.

                          Brand Name : Kaftrio

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          November 23, 2023

                          Lead Product(s) : Ivacaftor,Tezacaftor,Elexacaftor

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          05

                          2024 ACI Convention
                          Not Confirmed
                          2024 ACI Convention
                          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...

                          Brand Name : Trikafta

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          April 26, 2023

                          Lead Product(s) : Elexacaftor,Tezacaftor,Ivacaftor

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          06

                          2024 ACI Convention
                          Not Confirmed
                          2024 ACI Convention
                          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...

                          Brand Name : Trikafta

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          November 03, 2022

                          Lead Product(s) : Elexacaftor,Tezacaftor,Ivacaftor

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          07

                          2024 ACI Convention
                          Not Confirmed
                          2024 ACI Convention
                          Not Confirmed

                          Details : TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) real-world safety and effectiveness interim results show improved lung function and significant reductions in risk of pulmonary exacerbations, lung transplant and death for people with cystic fi...

                          Brand Name : Trikafta

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          June 10, 2022

                          Lead Product(s) : Ivacaftor,Tezacaftor,Elexacaftor

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          08

                          2024 ACI Convention
                          Not Confirmed
                          2024 ACI Convention
                          Not Confirmed

                          Details : TRIKAFTA is an oral medicine designed to increase quantity and function of CFTR protein at cell surface, is a prescription medicine used for treatment of cystic fibrosis in patients ages 6 years and older who have at least one copy of F508del mutation in...

                          Brand Name : Trikafta

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          April 20, 2022

                          Lead Product(s) : Elexacaftor,Tezacaftor,Ivacaftor

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          09

                          2024 ACI Convention
                          Not Confirmed
                          2024 ACI Convention
                          Not Confirmed

                          Details : TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) was approved by the Australian Therapeutic Goods Administration (TGA) in March 2021 based on the results of four global Phase 3 clinical trials, which included multiple Australian trial sites an...

                          Brand Name : Trikafta

                          Molecule Type : Small molecule

                          Upfront Cash : Undisclosed

                          March 27, 2022

                          Lead Product(s) : Elexacaftor,Tezacaftor,Ivacaftor

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved

                          Sponsor : Australian Pharmaceutical Benefits Scheme

                          Deal Size : Undisclosed

                          Deal Type : Agreement

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                          10

                          2024 ACI Convention
                          Not Confirmed
                          2024 ACI Convention
                          Not Confirmed

                          Details : EC approval for KAFTRIO (ivacaftor/tezacaftor/elexacaftor) in combination with ivacaftor has shown clinical benefit for people with CF ages 12 and above. This medicine is also approved in the regulatory authorities of New Zealand and Switzerland, where i...

                          Brand Name : Kaftrio

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          January 11, 2022

                          Lead Product(s) : Ivacaftor,Tezacaftor,Elexacaftor

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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