Vertex Says FDA Accepts For Priority Review SNDA For Use Of ORKAMBI In Children

(RTTNews) - Vertex Pharmaceuticals Inc. (VRTX) announced that the U.S. Food and Drug Administration has accepted for review a supplemental New Drug Application or sNDA for the use of ORKAMBI or lumacaftor/ivacaftor in people with cystic fibrosis or CF ages 6 to 11 who have two copies of the F508del mutation. The FDA granted Vertex's request for Priority Review of this sNDA, and a target review date of September 30, 2016 was set under the Prescription Drug User Fee Act or PDUFA.

The sNDA is based on data from an open label Phase 3 clinical safety study of ORKAMBI.

To support potential approval in the European Union, a six-month Phase 3 efficacy study of children ages 6 to 11 is ongoing. Vertex recently completed enrollment in this study and, pending data from the study, plans to submit a Marketing Authorization Application or MAA variation in the European Union in the first half of 2017 for children ages 6 to 11 who have two copies of the F508del mutation.

The primary endpoint of this efficacy study is the absolute change in lung clearance index or LCI. There are approximately 3,400 children ages 6 to 11 who have two copies of the F508del mutation in the European Union.