The U.S. Food and Drug Administration has approved the use of tezacaftor/ivacaftor (Symdeko®) for children with cystic fibrosis ages 6 to 11 with specific mutations.
Site Search
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.
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 U.S. Food and Drug Administration has approved the use of ivacaftor (Kalydeco®) for children as young as 6 months.
Today, Vertex Pharmaceuticals Inc. announced that the U.S. Food and Drug Administration has accepted its application to expand Trikafta® (elexacaftor/tezacaftor/ivacaftor) to include children ages 6-11 years old with cystic fibrosis who have at least one F508del or a mutation in the CFTR gene that is responsive based on in vitro data. The FDA has granted priority review of the application and has indicated that it will make a decision by June 8, 2021.
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 clinical trial data for Trikafta® (elexacaftor/tezacaftor/ivacaftor) in children 6-11 years old with cystic fibrosis.