traffic light



FDA approves Vertex’s TRIKAFTA®, SYMDEKO® and KALYDECO® for use in people with cystic fibrosis with certain rare mutations

Vertex Pharmaceuticals, a biotechnology company developing therapeutics for people with serious diseases, announced that TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor), SYMDEKO® (tezacaftor/ivacaftor and ivacaftor) and KALYDECO® (ivacaftor) received approvals from FDA to include additional responsive CFTR mutations in people with cystic fibrosis. These approvals allow more than 600 people with cystic fibrosis not previously eligible for these medicines an opportunity to potentially benefit from treatment.

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 KALYDECO is now approved for an additional 59 mutations, for a total of 97 KALYDECO-responsive mutations.

– Read the press release –