CF is a rare, progressive, multi-system genetic disease that affects the lungs, liver, GI tract, sinuses, sweat glands, pancreas and reproductive tract
Image: European Commission building. Photo: Courtesy of Sébastien Bertrand/Wikipedia.
Vertex Pharmaceuticals (Europe) has secured the European Commission (EC) approval for the label extension for KALYDECO (ivacaftor) to include the treatment of cystic fibrosis (CF) in children aged between 6 months and 12 months.
EC has approved KALYDECO for CF in infants with 5kg and more weight and with at least one of the following nine mutations, including G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R, in their cystic fibrosis transmembrane conductance regulator (CFTR) gene.
Vertex executive vice president and chief medical officer Reshma Kewalramani said: “Today’s approval is another milestone in our commitment to treat all people with CF as early in life as possible, given manifestations of CF are often present at birth.
“For the first time, children with CF in Europe as young as 6 months with certain mutations now have access to a medicine that treats the underlying cause of their disease.”
Label update of KALYDECO was based on the data from on Phase 3 ARRIVAL study
CF is a rare, progressive, multi-system genetic disease that affects the lungs, liver, GI tract, sinuses, sweat glands, pancreas and reproductive tract.
The disease is caused when a CFTR protein becomes defective or missing due to certain mutations in the CFTR gene. Children will have CF if they inherit two defective CFTR genes, one from each parent.
ivacaftor is an oral medicine, said to be the first medicine for treating people with specific mutations in the CFTR gene, and is also known as a CFTR potentiator.
The drug has been approved for people with CF, and specific mutations in the CFTR gene, in countries across North America, Europe and in Australia.
Vertex said that the label update is supported by data from the ongoing Phase 3 open-label safety study (ARRIVAL) in children aged less than 24 months with CF, and a CFTR gating mutation.
The study demonstrated a similar safety profile to the previous Phase 3 studies of older children and adults, and reached secondary endpoint of improvement in sweat chloride.
KALYDECO is already approved in Europe for CF patients aged 12 months and older and is also approved for patients aged 18 years and older, with R117H mutation in the CFTR gene.