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[{"orgOrder":0,"company":"NHS England","sponsor":"Vertex Pharmaceuticals","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Vertex Announces Expansion of Reimbursement Agreement With NHS England","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase IV","country":"","productType":"Small molecule","productStatus":"Approved","date":"June 2020","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase IV"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Vertex Pharmaceuticals Reports Positive Phase 3 Study Results for TRIKAFTA\u00ae in People Ages 12 and Older With Cystic Fibrosis","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase IV","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"July 2020","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase IV"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Vertex\u2019s Kaftrio Approved for Use in Europe and Made Accessible on the NHS","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"August 2020","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"FDA Accepts Vertex\u2019s Supplemental New Drug Applications for TRIKAFTA\u00ae , SYMDEKO\u00ae and KALYDECO\u00ae for Additional CFTR Mutations","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small 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Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"June 2021","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"French Health Authorities for the cystic fibrosis","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Vertex Announces National Reimbursement Agreement in France for KAFTRIO\u00ae for Eligible Cystic Fibrosis Patients","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"June 2021","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Spanish government","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Vertex Announces Reimbursement Agreement in Spain for 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Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"April 2021","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Australian Pharmaceutical Benefits Scheme","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Vertex Announces Reimbursement Agreement in Australia for TRIKAFTA\u00ae (elexacaftor\/tezacaftor\/ivacaftor and ivacaftor) for Patients With Cystic Fibrosis Ages 12 Years and Older With at Least One F508del Mutation in the CFTR Gene","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"March 2022","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not 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Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Vertex to Present Long-Term Data Demonstrating Significant Benefits of Treatment With CFTR Modulators at North American Cystic Fibrosis Conference (NACFC)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"October 2021","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Vertex Receives CHMP Positive Opinion for KAFTRIO\u00ae (Ivacaftor\/Tezacaftor\/Elexacaftor) in Combination With Ivacaftor in Children With Cystic Fibrosis Ages 6 Through 11","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small 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ivacaftor) in Children With Cystic Fibrosis Ages 6 Through 11 years With At Least One F508del Mutation","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"April 2022","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Vertex to Present Data Demonstrating Significant Benefits of Long-Term and Early Treatment With CFTR Modulators at the European Cystic Fibrosis Conference","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"June 2022","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Not 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2023","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Vertex Announces European Medicines Agency Validation for Marketing Authorization Application Extension for KAFTRIO\u00ae in Combination With Ivacaftor to Include People With Cystic Fibrosis and Responsive Rare Mutations","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"November 2023","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"European Commission Approves KAFTRIO\u00ae in Combination With Ivacaftor for the Treatment of Children With Cystic Fibrosis Ages 2 Through 5","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"November 2023","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Vertex Announces Positive Results From Pivotal Trials of Vanzacaftor\/Tezacaftor\/Deutivacaftor, Next-in-Class Triple Combination Treatment for Cystic Fibrosis","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"February 2024","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III"}]

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            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.

            Lead Product(s): Vanzacaftor,Tezacaftor,Deutivacaftor

            Therapeutic Area: Genetic Disease Product Name: VX-121

            Highest Development Status: Phase III Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable February 05, 2024

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            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.

            Lead Product(s): Ivacaftor,Tezacaftor,Elexacaftor

            Therapeutic Area: Genetic Disease Product Name: Kaftrio

            Highest Development Status: Approved Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable November 24, 2023

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            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.

            Lead Product(s): Ivacaftor,Tezacaftor,Elexacaftor

            Therapeutic Area: Genetic Disease Product Name: Kaftrio

            Highest Development Status: Approved Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable November 23, 2023

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            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 at the cell surface.

            Lead Product(s): Elexacaftor,Tezacaftor,Ivacaftor

            Therapeutic Area: Genetic Disease Product Name: Trikafta

            Highest Development Status: Approved Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable April 26, 2023

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            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 symptom and CFTR function.

            Lead Product(s): Elexacaftor,Tezacaftor,Ivacaftor

            Therapeutic Area: Genetic Disease Product Name: Trikafta

            Highest Development Status: Approved Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable November 03, 2022

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            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 fibrosis (CF).

            Lead Product(s): Ivacaftor,Tezacaftor,Elexacaftor

            Therapeutic Area: Genetic Disease Product Name: Trikafta

            Highest Development Status: Approved Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable June 10, 2022

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            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 CFTR gene.

            Lead Product(s): Elexacaftor,Tezacaftor,Ivacaftor

            Therapeutic Area: Genetic Disease Product Name: Trikafta

            Highest Development Status: Approved Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable April 20, 2022

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            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 and patients.

            Lead Product(s): Elexacaftor,Tezacaftor,Ivacaftor

            Therapeutic Area: Genetic Disease Product Name: Trikafta

            Highest Development Status: Approved Product Type: Small molecule

            Partner/Sponsor/Collaborator: Australian Pharmaceutical Benefits Scheme

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Agreement March 27, 2022

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            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 it is known as TRIKAFTA.

            Lead Product(s): Ivacaftor,Tezacaftor,Elexacaftor

            Therapeutic Area: Genetic Disease Product Name: Kaftrio

            Highest Development Status: Approved Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable January 11, 2022

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            Details:

            The agreement covers people with CF ages 12 years and older who have at least one copy of the F508del mutation, in the CFTR gene. KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with ivacaftor can be prescribed to eligible patients by treating physicians.

            Lead Product(s): Ivacaftor,Tezacaftor,Elexacaftor

            Therapeutic Area: Genetic Disease Product Name: Kaftrio

            Highest Development Status: Approved Product Type: Small molecule

            Partner/Sponsor/Collaborator: Spanish government

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Agreement November 19, 2021

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