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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 CFTR gene.


Lead Product(s): Lumacaftor,Ivacaftor

Therapeutic Area: Genetic Disease Product Name: Orkambi

Highest Development Status: ApprovedProduct 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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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: ApprovedProduct 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: ApprovedProduct 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: ApprovedProduct Type: Small molecule

Partner/Sponsor/Collaborator: FSIO

Deal Size: Undisclosed Upfront Cash: Undisclosed

Deal Type: Agreement April 21, 2020

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