Vertex Announces it will Submit Cystic Fibrosis Medicines ORKAMBI (lumacaftor/ivacaftor) as well as SYMKEVI (tezacaftor/ivacaftor) to be Used in Combination with ivacaftor, to the Scottish Medicines Consortium for Appraisal

Vertex Pharmaceuticals (Europe) Limited today announced that, following constructive discussions with the Scottish Government, it will submit ORKAMBI (lumacaftor/ivacaftor) as well as SYMKEVI (tezacaftor/ivacaftor) to be used in combination with ivacaftor, to the Scottish Medicines Consortium (SMC) for appraisal. If accepted by the SMC for use on the NHS in Scotland, eligible patients with cystic fibrosis (CF) in Scotland could have access to these precision medicines in 2019.

“Our recent conversations with the Scottish Government, and the SMC’s orphan medicines process have provided important flexibility for evaluating precision medicines, such as cystic fibrosis transmembrane conductance regulator (CFTR) modulators. Their methods reflect the innovative nature of medicines that have the potential to extend life for patients with rare diseases, like CF,” said Ludovic Fenaux, Senior Vice President, Vertex International. “We are hopeful that, through this process, all eligible patients in Scotland could have access to our medicines soon.”

While the SMC reviews the submissions, clinicians could apply for access to lumacaftor/ivacaftor as well as tezacaftor/ivacaftor in combination with ivacaftor for individual patients based on clinical need via the Peer Approved Clinical System (PACS) Tier 2 process for ‘individual access to medicines not yet generally available on the NHS’. Vertex and the Scottish Government have also agreed to a confidential discount which will be applied to applications to the PACS Tier 2 process.

Approximately 900 people in Scotland live with CF.¹ In the UK, the median age of death is 31 years.² NHS Scotland estimates that one in 24 Scots have a CFTR mutation which, if carried by both parents, would lead to a child being born with CF.³

Should the appraisal be positive, patients in Scotland will join those in other countries around the world where all those eligible have access to lumacaftor/ivacaftor, including Austria, Australia, Denmark, Germany, the Republic of Ireland, Italy, the Netherlands, Sweden and the U.S.

About cystic fibrosis
Cystic fibrosis (CF) is a rare, life-shortening genetic disease affecting approximately 75,000 people in North America, Europe and Australia.⁴

CF is caused by a defective or missing cystic fibrosis transmembrane conductance regulator (CFTR) protein resulting from mutations in the CFTR gene. Children must inherit two defective CFTR genes — one from each parent — to have CF. There are approximately 2,000 known mutations in the CFTR gene.⁵ Some of these mutations, which can be determined by a genetic test, or genotyping test, lead to CF by creating non-working or too few CFTR proteins at the cell surface. The defective function or absence of CFTR protein results in poor flow of salt and water into and out of the cell in a number of organs. In the lungs, this leads to the build-up of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage in many patients that eventually leads to death.⁶