FDA Approves Orkambi for Expanded Use in Children with Cystic Fibrosis


With this approval, about 300 children between 12 months and 24 months will be eligible for treatment with Orkambi.

The FDA has approved expanded the use of Vertex Pharmaceuticals’ Orkambi (lumacaftor/ivacaftor) to include children with cystic fibrosis (CF) ages 12 months to <24 months with two copies of the F508del mutation. Orkambi was previously approved for use in patients ages 2 years and older with two copies of the F508del mutation.

Cystic fibrosis is a rare, life-shortening genetic disease that affects more than 83,000 people globally. It is a progress disease that can affects the lungs, liver, pancreas, GI tract, sinuses, sweat glands and reproductive tract. Cystic fibrosis is caused by a defective and/or missing CFTR protein resulting from certain mutations in the CFTR gene, and children must inherit two defective genes to have cystic fibrosis.

Carmen Bozic, M.D.

Carmen Bozic, M.D.

“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,” Carmen Bozic, M.D., executive vice president, global medicines development and medical affairs, and chief medical officer of Vertex, said in a press release.

The approval in children ages 12 months to <24 months is based on a 24-week, phase 3 study in 46 children ages 1 to less than 2 years with the two copies of the F508del mutation. Orkambiwas generally well tolerated, and the safety profile and pharmacokinetics were similar to that observed in studies in patients ages 2 years and older. Additional study results, including reductions in sweat chloride concentration, suggest the potential for CF disease modification with the use of Orkambi.

Orkambi as first approved in 2015 in the United States and is now available in more than 30 countries. The average wholesale price of Orkambi is $26,333.12 for 112 tablets, or $235.11 per tablet, according to Vertex. It is also available as oral granules for reconstitution.

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