BRIEF—FDA approval for Orkambi in infants under two years

The US Food and Drug Administration (FDA) on Friday approved expanded use of Orkambi (lumacaftor/ivacaftor) to include children with cystic fibrosis (CF) ages 12 to <24 months who are homozygous for the F508del mutation (F/F genotype) in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

Marketed by US drugmaker Vertex Pharmaceuticals, Orkambi was previously approved by the FDA for use in people with CF ages 2 years and older with two copies of the F508del mutation.

“Treating children with cystic fibrosis as early in life as possible is critically important, because early treatment has the potential to slow the progression of this devastating disease,” said Dr Carmen Bozic, executive vice president, global medicines development and medical affairs, and chief medical officer, Vertex, adding: “Today’s approval is another important step on our journey to reach people of all ages living with cystic fibrosis who may benefit from our medicines.”

Orkambi was first approved in 2015 in the USA and is now available in more than 30 countries.