1 September 2020 - More than 600 people with certain rare cystic fibrosis mutations could become newly eligible for Trikafta, Symdeko or Kalydeco.

Vertex Pharmaceuticals today announced the U.S. FDA accepted three supplemental new drug applications for Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor), Symdeko (tezacaftor/ivacaftor and ivacaftor) and Kalydeco (ivacaftor). 

These regulatory submissions are intended to expand the labels for Trikafta, Symdeko and Kalydeco to include additional rare CFTR mutations, allowing people with cystic fibrosis not previously eligible for these medicines an opportunity to benefit from treatment that targets the underlying cause of their disease. In addition, these regulatory submissions may also allow certain people with cystic fibrosis who are currently eligible for Kalydeco to become eligible for Symdeko or Trikafta and certain people currently eligible for Symdeko may become eligible for Trikafta. 

The FDA has assigned a Prescription Drug User Fee Act target action date of 30 December 2020.

Read Vertex Pharmaceuticals press release