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.
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The U.S. Food and Drug Administration has approved the use of ivacaftor (Kalydeco®) for children as young as 6 months.
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.”
The Cystic Fibrosis Foundation awarded up to $5.1 million to TB Alliance to advance a drug discovery program in partnership with Johns Hopkins University. The effort will identify compounds with the potential to treat nontuberculous mycobacteria infections that are increasingly found among people with CF.
Today, Vertex Pharmaceuticals Inc. released the initial Phase 3 clinical trial data for one of two next-generation, triple-combination modulators currently being tested.
Vertex Pharmaceuticals Inc. today announced that it will pursue FDA approval for the triple-combination therapy VX-445 (elexacaftor) plus tezacaftor/ivacaftor (Symdeko®).
Today, Vertex released positive preliminary clinical trial results for the potential triple-combination therapy VX-445 plus tezacaftor/ivacaftor (Symdeko®).
The Institute for Clinical and Economic Review (ICER) recently posted a draft scoping document to outline their planned assessment of the clinical effectiveness and value of the triple combination (also known as elexacaftor/tezacaftor/ivacaftor). The document is open for public comment through October 21.
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.