EU label for Vertex's cystic fibrosis drug Orkambi expanded again to include even younger children

Vertex Pharmaceuticals on Monday announced that the European Commission expanded approval for Orkambi (lumacaftor/ivacaftor) to include treatment of children with cystic fibrosis (CF) aged two years to five years who are homozygous for the F508del mutation. The drug received a positive opinion for this latest indication last November by the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP). 

Orkambi was initially approved in Europe in 2015 for patients with CF ages 12 years and older who have two copies of the F508del mutation and was later expanded to include children ages six to 11 years. 

Vertex noted that Orkambi is the only approved medicine in Europe to treat the underlying cause of CF for the approximately 1500 children aged two to five years with two copies of the F508del mutation. The latest decision "brings us one step closer to our goal of bringing treatment to all people living with CF," commented Reshma Kewalramani, chief medical officer and head of global medicines development and medical affairs at Vertex. "By treating the underlying cause of disease early, we can potentially modify its course and offer patients the chance of improved outcomes," Kewalramani added. 

According to Vertex, the label update is based on data from a Phase III trial of 60 patients that showed treatment with Orkambi was "generally well tolerated for 24 weeks, with a safety profile in these paediatric patients generally consistent with that in patients aged six years and older." US regulators expanded approval of Orkambi last year to also include children ages two through five years. 

Meanwhile, the EU approved Vertex's Kalydeco (ivacaftor) last November for patients with CF aged 12 months to under 24 months who have at least one of nine specific mutations in their CFTR gene. The drug had already been cleared in Europe to treat CF in patients aged two years and older who have one of the nine mutations, as well as for adults who have an R117H mutation in the CFTR gene. 

European regulators also recently approved Vertex's Symkevi (tezacaftor/ivacaftor) in a combination regimen with Kalydeco for patients aged 12 and older who are either homozygous for the F508del mutation or have one copy of the F508del mutation and a copy of one of 14 other mutations in which the CFTR protein shows residual activity. 

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