Vertex' Trikafta case gets boost as EMA validates MAA

US drugmaker Vertex Pharmaceuticals (Nasdaq: VRTX) saw progress with its triple-combination cystic fibrosis (CF) treatment on two fronts on Thursday.

Phase III results on Trikafta (elexacaftor/tezacaftor/ivacaftor/ivacaftor), which was last month approved in the USA for patients aged 12 years and older who have at least one copy of the F508del mutation in the CF transmembrane conductance regulator (CFTR) gene, have been concurrently published in The New England Journal of Medicine and The Lancet.

Data highlighting primary and secondary endpoints from a 24-week study in 403 people with one F508del mutation and one minimal function mutation in the CFTR gene were published in NEJM.