The approval allows Kalydeco to be used for the treatment of children and adolescents with CF aged between six months and older with a weight of at least 5kg, who have the R117H mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
Ivacaftor, known as a CFTR potentiator, is an oral medication that helps to place CFTR proteins at the cell surface open longer to enhance the transport of salt and water across the cell membrane, helping to hydrate and clear mucus from the airways.
CF is a rare, multi-system and genetic disease that is said to affect the lungs, liver, GI tract, sinuses, sweat glands, pancreas and reproductive tract.
Vertex will also coordinate with all other relevant government authorities to secure access for eligible patients.
Kalydeco is already approved in Europe for the treatment of people with CF ages 18 and older with the R117H mutation and in infants aged six months and older weighing at least 5kg who have one of the mutations in the CFTR gene such as G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R.
Vertex CEO and president Dr Reshma Kewalramani said: “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.
“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 September 2019, Vertex agreed to acquire US-based biotech company Semma Therapeutics for $950m (£784.66m) in an all-cash deal with an aim to develop curative cell-based treatments for type 1 diabetes.