Vertex announces reimbursement agreement in Australia for ORKAMBI

Vertex Pharmaceuticals announced the finalization of an agreement with the Australian Government that allows for reimbursement of ORKAMBI (lumacaftor/ivacaftor) for people ages six and over with cystic fibrosis (CF) who have two copies of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. This agreement means lumacaftor/ivacaftor will be listed on the Pharmaceutical Benefits Scheme (PBS) from 1 October and follows a positive recommendation from the Pharmaceutical Benefits Advisory Committee (PBAC). A pathway to access for future Vertex CF medicine, tezacaftor/ivacaftor, has been established as part of this process.

"We are delighted that lumacaftor/ivacaftor will be made available to eligible CF patients in Australia," said Stuart Arbuckle, Executive Vice President and Chief Commercial Officer at Vertex. "We would like to recognize the PBAC for seeing the value of this medicine and acknowledge the Department of Health and the Minister for Health for their active engagement and willingness to finalize the agreement rapidly."

Australians with CF join thousands of patients worldwide who are already receiving lumacaftor/ivacaftor. Beyond Australia, countries where lumacaftor/ivacaftor is reimbursed include Austria, Denmark, Germany, Ireland, Italy, the Netherlands, Sweden and the U.S.

Cystic fibrosis is a rare, life-shortening genetic disease affecting approximately 75,000 people including countries within North America and Europe, as well as Australia.

ORKAMBI is a combination of lumacaftor, which 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, and ivacaftor, which is designed to enhance the function of the CFTR protein once it reaches the cell surface. Lumacaftor/ivacaftor is available as tablets and is typically taken twice per day.

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