Vertex Pharmaceuticals says the European Commission has granted approval of the label extension for Kalydeco, to include the treatment of patients with cystic fibrosis (CF) aged 12 to 24 months.
The move is based on data from the ongoing Phase III safety study (ARRIVAL) of children with the disease aged 12 to 24 months, who have one of 10 mutations in the CFTR gene.
The data show a safety profile consistent with that observed in previous studies involving older children and adults, the firm noted.
Kalydeco (ivacaftor) is the first and only approved medicine in Europe to treat the underlying cause of cystic fibrosis in such young patients.
It is already available in the UK for patients aged two years and older who have one of the nine following mutations in the CFTR gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R. It is also approved for the treatment of CF in patients aged 18 years and older who carry an R117H mutation in the CFTR gene.
“With this important milestone, we are a step closer to enabling physicians to treat the underlying cause of CF earlier than ever, helping to improve clinical outcomes in children as young as 12 months old,” said Simon Lem, VP regional general manager Northern Europe for Vertex.
“It is now our priority to ensure that eligible patients in the UK are able to access this medicine as soon as possible and we will continue to work closely with reimbursement authorities across the UK.”
CF accounts for 9,500 hospital admissions and over 100,000 hospital bed days every year; a third of these are used by children under 15 years old. Currently, there is no cure for CF and half of people in the UK with the condition die before they are 31.