Vertex’s Kalydeco scores another label extension in EU

Vertex has scored approval from the European Commission to extend the label for Kalydeco (ivacaftor) to include the treatment o infants with cystic fibrosis (CF) aged four months or older.

To be eligible for Kalydeco treatment, infants must weigh at least 5kg and have the R117H mutation or one of the following gating (class III) mutations in the CFTR gene – G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R.

The drug is already approved in Europe for people with CF aged six months or older and weighing at least 5kg, with the same mutations in the CFTR gene listed above.

The latest label extension is based on data from a cohort in the 24-week phase III open-label safety study – ARRIVAL – consisting of six children with CF aged four months to less than six months who have eligible gating mutations.

“Our very first CFTR modulator, Kalydeco, was first approved eight years ago, for certain CF patients ages 6 years and older. With today’s (5 November) approval, babies as young as four months are eligible and we believe early treatment is important in managing CF,” said Reshma Kewalramani, chief executive officer and president, Vertex.

“Today’s approval is a testament to our commitment to keep going until all people with CF have a treatment option,” he added.

According to Vertex, over 10,500 people in the UK have CF – the second highest number across the world.

Kalydeco is an oral medicine designed to keep CFTR proteins at the cell surface open longer, to improve the transport of salt and water across the cell membrane.

This helps to hydrate and clear mucus from the airways, targeting the underlying cause of CF.