The U.S. Food and Drug Administration has approved the use of Kalydeco® (ivacaftor) for infants with cystic fibrosis as young as four months who have certain mutations.
Today, Vertex Pharmaceuticals Inc. announced positive Phase 3 clinical trial data for Trikafta® (elexacaftor/tezacaftor/ivacaftor) in children 6-11 years old with cystic fibrosis.
Vertex Pharmaceuticals Inc. has submitted three supplemental New Drug Applications to the U.S. Food and Drug Administration (FDA) to expand eligibility for Trikafta® (elexacaftor/tezacaftor/ivacaftor), Symdeko® (tezacaftor/ivacaftor and ivacaftor) and Kalydeco® (ivacaftor) to additional rare CFTR mutations.
Today, Vertex Pharmaceuticals Inc. announced positive Phase 3 study results for Trikafta® (elexacaftor/tezacaftor/ivacaftor) for people with cystic fibrosis ages 12 years and older who have one copy of the F508del mutation and one gating or residual function mutation.
The Cystic Fibrosis Foundation awarded nearly $700,000 to Calibr, the drug discovery and development arm of Scripps Research, to screen for a compound that could be used to treat Burkholderia cepacia complex infections.
The Cystic Fibrosis Foundation today announced an unprecedented acceleration and expansion of its research, care and patient programs for the CF community. This action is possible as a result of the sale of the Foundation's royalty rights to CF treatments developed by Vertex Pharmaceuticals Inc. The Foundation received $3.3 billion from the sale to Royalty Pharma.
CF Foundation President and CEO Robert J. Beall, Ph.D. testified today before the House of Representatives' Energy and Commerce Committee at a hearing entitled, “21st Century Cures: Incorporating the Patient Perspective.”