‘Vertex is disappointed that the Scottish Medicines Consortium (SMC) has not recommended ORKAMBI for people with cystic fibrosis ages 12 and older who have two copies of the F508del mutation or KALYDECO for children with cystic fibrosis (CF) ages two to five who have gating mutations for use within NHS Scotland,’ said Simon Bedson, Senior Vice President and International General Manager at Vertex, following news of the decision.
‘However, the Scottish government has established the New Medicines Fund to provide access to medicines for rare diseases; KALYDECO has been available for people with CF ages six and older who have the G551D mutation via this fund since 2013. Vertex firmly believes that the New Medicines Fund is the appropriate means for eligible people with CF to access ORKAMBI and KALYDECO.
‘We urge the Scottish government to collaborate with all stakeholders to provide access to both medicines via the New Medicines Fund as quickly as possible.’
CF is a rare, life-shortening genetic disease that causes progressive damage to a number of organs throughout the body. In Scotland, approximately half of people with CF die by the age of 26. According to the 2014 UK CF registry, there are approximately 230 people eligible for ORKAMBI and five children ages two to five eligible for KALYDECO in Scotland.
‘Vertex remains committed to continuing to work with the Scottish government to make ORKAMBI and KALYDECO available to all eligible patients,’ added Simon.
