EU regulators have expanded the treatment scope of Vertex’ Kalydeco (ivacaftor) to include a new subset of young patients with cystic fibrosis (CF).
Doctors can now also prescribe the treatment for children and adolescents with CF, aged six months and older and weighing at least 5kg, who have the R117H mutation in the CF transmembrane conductance regulator (CFTR) gene – the most common residual function mutation underlying the condition.
Kalydeco (ivacaftor) will be immediately available to additional eligible patients in Germany and shortly in countries where respective long-term reimbursement agreements have been previously secured, Vertex said, adding that it will also ‘work closely’ with all other relevant government authorities ‘to secure access for eligible patients as quickly as possible’.
In Europe, the drug is already approved for the treatment of people with CF ages 18 and older with the R117H mutation, and in infants ages 6 months and older weighing at least 5kg who have one of the following mutations in the CFTR gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R.
“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,” said Reshma Kewalramani, Vertex’ chief executive and president.
The label extension means that around 500 young patients in Europe, ‘who have long awaited a treatment option, are now eligible for Kalydeco,” she added.
