Vertex will submit CF treatment Orkambi and Symkevi combination in Scotland

Vertex Pharmaceuticals 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 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 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 SystemTier 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.

https://thefly.com/landingPageNews.php?id=2839409