The FDA has approved Orkambi (lumacaftor/ivacaftor, Vertex Pharmaceuticals) for use in children 6 through 11years of age with cystic fibrosis (CF) who have two copies of the F508del mutation. People with this mutation represent the largest subset of those with CF. Orkambi is the only medication that treats the underlying cause of CF for people with this mutation.
The treatment was previously approved by the FDA for use in people 12 years of age and older with two copies of the F508del mutation. With the new approval, approximately 11,000 people with CF are eligible for treatment with Orkambi in the United States.
The FDA’s decision was based on data from an open-label phase 3 safety study of Orkambi presented at the 39th European Cystic Fibrosis Society Conference in June 2016. These findings will be presented in October at the 30th Annual North American Cystic Fibrosis Conference in Orlando, Florida.
Orkambi is a combination of lumacaftor, which is designed to increase the amount of mature protein at the cell surface by targeting the processing and trafficking defect of the F508del CFTR protein, and ivacaftor, which is designed to enhance the function of the CFTR protein once it reaches the cell surface. In patients 6 through 11 years of age, two Orkambi tablets (each containing lumacaftor 100 mg and ivacaftor 125 mg) are taken orally every 12 hours––once in the morning and once in the evening–with fat-containing food.
Orkambi is used for the treatment of CF in patients 6 years of age and older who have two copies of the F508del mutation (F508del/F508del) in their CFTR gene. The treatment should be used only in these patients. It is not known whether Orkambi is safe and effective in children younger than 6 years of age.
Vertex initiated its CF research program in 2000 as part of a collaboration with Cystic Fibrosis Foundation Therapeutics, Inc., the nonprofit drug discovery and development affiliate of the Cystic Fibrosis Foundation. Orkambi was discovered by Vertex as part of this collaboration.
Source: Vertex Pharmaceuticals; September 28, 2016.