The European Committee for Medicinal Products for Human Use (CHMP) has recommended the approval of Orkambi (lumacaftor/ivacaftor) as a treatment for children ages 2-5 with cystic fibrosis (CF) due to F508del mutations, Vertex Pharmaceuticals announced.
CHMP’s positive opinion will be taken into consideration by the European Medicines Agency, which will now decide if the Orkambi label should be extended to include these younger patients.
Orkambi is currently approved in Europe to treat CF patients ages 6 and older who have the F508del mutation in the CFTR gene, which is defective in CF. If approved, Orkambi will become the first treatment available in Europe to treat approximately 1,500 young children whose CF is caused by two copies of the F508del mutation.
“Cystic fibrosis is a chronic, progressive disease and it is important to treat early to ensure the best possible outcomes for patients,” Reshma Kewalramani, MD, executive vice president of global medicines development and medical affairs and chief medical officer at Vertex, said in a press release.
“[This] announcement brings us one step closer to providing more young children with a treatment that addresses the underlying cause of their disease,” Kewalramani added. “It also marks another important milestone on our path towards our goal of treating every person with this rare and life-limiting disease.”
Vertex’s request to expand Orkambi’s marketing authorization application in Europe was based on clinical data from a Phase 3 open-label trial (NCT03125395), which evaluated the treatment’s safety in about 60 CF patients ages 2 and older who had two copies of the F508del mutation. The children received Orkambi every 12 hours, according to their age and weight.
Results showed that the treatment was well-tolerated and generally safe for 24 weeks, with children ages 2 to 5 showing a similar safety profile to those 6 and older. The most common adverse event reported was cough, affecting 63% of the children in the group. Still, most of the adverse events reported were mild or moderate in severity.
In addition, efficacy data at week 24 revealed that Orkambi could effectively reduce the levels of sweat chloride — a hallmark sign of the disease — in these children. Improvements in growth parameters, such as weight and stature, were also observed.
In August, the U.S. Food and Drug Administration extended the therapy’s label also for use in children ages 2-5.