New Phase III Trikafta data supports sNDA Regulatory submissions

Vertex Pharmaceuticals (Nasdaq: VRTX) has announced the completion of a global Phase III study of Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor) in children ages six through 11 years old with cystic fibrosis (CF) who have either two copies of the F508del mutation or one copy of the F508del mutation and one minimal function mutation.

Based on the results, the company says it will submit a supplemental New Drug Application (sNDA) to the US Food and Drug Administration in the fourth quarter of 2020, with additional global regulatory submissions to follow.

“Our aim is to extend eligibility to all patients who may benefit from this transformative medicine, and the positive results from the study in children ages 6 through 11 years old allows us to take another step forward toward this goal,” said Dr Carmen Bozic, executive vice president, global medicines development and medical affairs, and chief medical officer at Vertex, adding: “We are looking forward to filing an sNDA in the coming months and bringing Trikafta to younger people with CF.”