Vertex announces FDA approvals of Trikafta, Symdeko and Kalydeco for use in people with cystic fibrosis with certain rare mutations.

Vertex Pharmaceuticals Incorporated announced the FDA expanded the eligibility for Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor) to include people with cystic fibrosis (CF) ages 12 years and older with certain mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that are responsive to Trikafta based on in vitro data. Symdeko (tezacaftor/ivacaftor and ivacaftor) and Kalydeko (ivacaftor) also received approvals to include additional responsive mutations in people with CF ages 6 years and older and age 4 months and older, respectively. These approvals allow more than 600 people with CF not previously eligible for these medicines an opportunity to potentially benefit from treatment that targets the underlying cause of their disease. Trikafta was previously approved for people with at least one F508del mutation and is now approved for 177 additional mutations; Symdeko is now approved for 127 additional mutations, for a total of 154 Symdeko-responsive mutations; and Kalydeko is now approved for an additional 59 mutations, for a total of 97 Kalydeko-responsive mutations. In addition, for certain people with CF who are currently eligible for Kalydeko, this approval allows them to also be eligible for Symdeko or Trikafta and similarly, for those who are currently eligible for Symdeko, this approval allows them to also be eligible for Trikafta