Vertex Receives CHMP Positive Opinion For ORKAMBI - Quick Facts

(RTTNews) - Vertex Pharmaceuticals Inc. (VRTX) announced the European Union Committee for Medicinal Products for Human Use (CHMP) issued a positive opinion recommending extension of the Marketing Authorization for ORKAMBI (lumacafator/ivacaftor) to children with cystic fibrosis ages 6 through 11 who have two copies of the F508del mutation.

RKAMBI 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. It is an oral pill taken every 12 hours - once in the morning and once in the evening.