Cystic Fibrosis Ireland welcomes the announcement today (10 June) that the European Commission has granted approval of the extension for Kalydeco (ivacaftor) to include the treatment of children and young people with cystic fibrosis (CF), ages 6 months and older and weighing at least 5 kg, who have the R117H mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene – one of the more common mutations in CF patients in Ireland.

In Ireland, the medicine will be available to approximately 35 patients within weeks due to the innovative 'pipeline' or 'portfolio' reimbursement agreement that was signed between Vertex and the HSE in 2017. This agreement means that once an extension to an existing CFTR drug has been agreed by the European Commission, it will become available in Ireland without having to go through the National Centre for Pharmacoeconomics (NCPE).

Reas the press release from Vertex below.

Vertex Announces European Commission Approval for KALYDECO® (ivacaftor) for Children and Adolescents With Cystic Fibrosis Between the Ages of 6 Months and 18 Years With the R117H Mutation in the CFTR Gene

-KALYDECO (ivacaftor) is the first and only approved medicine in Europe to treat the underlying cause of cystic fibrosis in patients with the R117H mutation, the most common residual function mutation, in children as young as 6 months of age-

-Approximately 35 eligible patients in Ireland will be able to access this medicine within weeks due to the innovative long-term reimbursement agreement that was signed between Vertex and the Health Service Executive (HSE) in 2017-

DUBLIN – 10th June, 2020– Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the European Commission has granted approval of the label extension for Kalydeco (ivacaftor) to include the treatment of children and adolescents with cystic fibrosis (CF), ages 6 months and older and weighing at least 5 kg, who have the R117H mutation in the cystic fibrosis transmembrane conductance regulator(CFTR) gene – one of the more common mutations in CF patients in Ireland[1].  In Ireland, the medicine will be available to approximately 35 eligible patients within weeks due to the innovative long-term reimbursement agreement that was signed between Vertex and the HSE in 2017.

“A little over eight years ago, Kalydeco was approved as the first and only medicine to treat the underlying cause of cystic fibrosis in patients with specific mutations,” said Reshma Kewalramani, M.D., Chief Executive Officer and President at Vertex. “Since then, it’s been our goal to ensure that as many people with CF as possible are able to benefit from our treatments, and today’s label extension means that approximately 500 young patients in Europe, who have long awaited a treatment option, are now eligible for Kalydeco.”

In Europe, the medicine is already approved for the treatment of people with CF ages 18 and older with the R117H mutation, and in infants ages 6 months and older weighing at least 5 kg who have one of the following mutations in the CFTR gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R.

ENDS