Vertex Pharma: CHMP Adopts Positive Opinion For ORKAMBI - Quick Facts

(RTTNews) - Vertex Pharmaceuticals (VRTX) Limited announced the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion for ORKAMBI (lumacaftor/ivacaftor) for the treatment of people with cystic fibrosis aged 2 to 5 years old who have two copies of the F508del mutation, the most common form of the disease. Lumacaftor/ivacaftor is already approved in Europe for the treatment of CF in patients aged 6 and older who have two copies of the F508del mutation.

Lumacaftor/ivacaftor 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.