The FDA has approved Vertex Pharma’s Orkambi (lumacaftor 200 mg/ivacaftor 125 mg), the first drug directed at treating cystic fibrosis patients 12 years and older with two copies of the F508del mutation.

The F508del mutation, which causes the production of an abnormal protein that disrupts how water and chloride are transported in the body, is the most common cause of CF. People who have two copies of the F508del mutation, one inherited from each parent, account for approximately half of the 30,000 CF population in the US.

The safety and efficacy of Orkambi was studied in two double-blind, placebo-controlled clinical trials of 1,108 participants with CF who were 12 years and older with the F508del mutation. In both studies, participants with CF who took Orkambi, two pills taken every 12 hours, demonstrated improved lung function compared to those who took placebo.

The efficacy and safety of Orkambi have not been established in patients with CF other than those with the F508del mutation. If a patient’s genotype is unknown, an FDA cleared CF mutation test should be used to detect the presence of the F508delmutation on both alleles of the CFTR gene.

The most common side effects of Orkambi include shortness of breath, upper respiratory tract infection, nausea, diarrhea, and rash. Women who took Orkambi also had increased menstrual abnormalities such as increased bleeding.

More information is available at Vertex Pharmaceuticals’ website.