Posted by Michael Wonder on 21 Dec 2020
Vertex announces FDA approvals of Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor), Symdeko (tezacaftor/ivacaftor and ivacaftor) and Kalydeco (ivacaftor) for use in people with cystic fibrosis with certain rare mutations
21 December 2020 - More than 600 people with certain rare cystic fibrosis mutations are now eligible for Trikafta, Symdeko or Kalydeco.
Vertex Pharmaceuticals today announced the U.S. FDA expanded the eligibility for Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor) to include people with cystic fibrosis ages 12 years and older with certain mutations in the cystic fibrosis transmembrane conductance regulator gene that are responsive to Trikafta based on in vitro data.
Symdeko (tezacaftor/ivacaftor and ivacaftor) and Kalydeco (ivacaftor) also received approvals to include additional responsive mutations in people with cystic fibrosis ages 6 years and older and age 4 months and older, respectively. These approvals allow more than 600 people with cystic fibrosis not previously eligible for these medicines an opportunity to potentially benefit from treatment that targets the underlying cause of their disease.
