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[{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"FSIO","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Vertex Announces Innovative Reimbursement Agreement in Switzerland for ORKAMBI\u00ae and SYMDEKO\u00ae for Eligible Cystic Fibrosis Patients","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"April 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":"Vertex Announces U.S. FDA Approval for ORKAMBI\u00ae (lumacaftor\/ivacaftor) in Children With Cystic Fibrosis Ages 12 to <24 months","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"September 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 Receives CHMP Positive Opinion for ORKAMBI\u00ae (lumacaftor\/ivacaftor) in Children With Cystic Fibrosis Ages 1 to <2 Years Old","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"April 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 ORKAMBI (lumacaftor\/ivacaftor) for the Treatment of Children With Cystic Fibrosis Ages 1 to <2 Years Old","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"July 2023","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Approved"}]

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            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 CFTR gene.

            Lead Product(s): Lumacaftor,Ivacaftor

            Therapeutic Area: Genetic Disease Product Name: Orkambi

            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 July 05, 2023

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            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 CFTR gene.

            Lead Product(s): Lumacaftor,Ivacaftor

            Therapeutic Area: Genetic Disease Product Name: Orkambi

            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 27, 2023

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            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-CFTR protein.

            Lead Product(s): Lumacaftor,Ivacaftor

            Therapeutic Area: Genetic Disease Product Name: Orkambi

            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 September 02, 2022

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

            Lead Product(s): Lumacaftor,Ivacaftor

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Approved Product Type: Small molecule

            Partner/Sponsor/Collaborator: FSIO

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Agreement April 21, 2020

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