EU approves Orkambi (lumacaftor/ivacaftor) for cystic fibrosis F508del mutation- Vertex Pharmaceuticals

Vertex Pharmaceuticals announced that the European Commission has granted Marketing Authorization for Orkambi (lumacaftor/ivacaftor), the first medicine to treat the underlying cause of cystic fibrosis (CF) in people ages 12 and older who have two copies of the F508del mutation.

The approval is based on previously announced data from two 24-week global Phase III studies, TRAFFIC and TRANSPORT, and additional interim 24-week data from the subsequent extension study, PROGRESS, in people ages 12 and older who have two copies of the F508del mutation and were already being treated with standard-of-care medicines.

In the TRAFFIC and TRANSPORT studies, which enrolled more than 1,100 patients, those treated with the combination of lumacaftor and ivacaftor experienced significant improvements in lung function. Patients also experienced improvements in body mass index (BMI) and reductions in pulmonary exacerbations (acute lung infections) including those requiring hospitalizations and intravenous antibiotic use. Interim data from PROGRESS showed that these improvements were sustained through 48 total weeks of treatment (24 weeks in TRAFFIC/TRANSPORT + 24 weeks in PROGRESS). In addition, the pattern and magnitude of response observed after the initiation of combination treatment across all patients who received placebo in TRAFFIC and TRANSPORT and subsequently received a combination regimen in PROGRESS were similar to those seen among patients who received a combination regimen in TRAFFIC and TRANSPORT. The combination of lumacaftor and ivacaftor was generally well tolerated in all three studies.