DRUG DEVELOPMENT PIPELINE

Back to the Drug Development Pipeline

Lumacaftor + ivacaftor (Orkambi®)

To Patients

To Patients

Therapeutic Approach

Restore CFTR Function

Lumacaftor and ivacaftor (Orkambi®) is a combination therapy combining lumacaftor, which is designed to fix the defective CFTR protein so that it can move to the proper place on the cell surface, with ivacaftor, which helps improve the function of the protein as a chloride channel on the cell surface.

Status

In the US, the FDA has approved the use of Orkambi® in people with CF who have two copies of the F508del CFTR mutation and are 6 years and older. A phase 3 study of Orkambi® in children aged 2-5 years with two copies of the F508del CFTR mutation is currently underway.

In Australia, Orkambi is awaiting PBAC reommendation for reimbursement.

Sponsor

In the US, Vertex Pharmaceuticals developed this drug with funding from Cystic Fibrosis Foundation Therapeutics (CFFT). The drug development was conducted within CFFT's Therapeutics Development Network.

Contact us about Lumacaftor + ivacaftor (Orkambi®) >