NEW YORK – The European Commission expanded the indication for Vertex Pharmaceuticals' cystic fibrosis drug Kalydeco (ivacaftor) to include infants as young as 1 month old, the biotechnology company said Friday.
Cystic fibrosis is a rare and progressive multi-organ disease caused by mutations in the CFTR gene that result in abnormally functioning CFTR proteins and poor flow of salt and water in and out of cells.
Kalydeco, which is already approved for patients ages 4 months and older in the EU, is an oral medication for those with certain mutations in the CFTR gene. Kalydeco is designed to facilitate CFTR proteins' ability to transport salt and water across the cell membrane.
"Today's approval is an important milestone for the cystic fibrosis community," Carmen Bozic, Vertex's chief medical officer and executive VP of global medicines development and medical affairs, said in a statement. "Treating CF early in life can potentially slow the progression of the disease, which is why it is so important to start treatment from a very young age."
With the EC's latest approval, eligible patients in Austria, the Czech Republic, Denmark, Ireland, Norway, Sweden, and the Netherlands will soon have access to the expanded indication of Kalydeco based on existing access agreements. Vertex said it will continue to work with authorities across the EU to ensure access to eligible patients.
Kalydeco is also available for patients as young as 1 month old in the UK and the US, based on a Phase III trial that showed that the drug is as safe in younger children as it is in older children and adults.