Positive results from a study of ivacaftor (Kalydeco®) in children ages 1 to 2 were announced today.
Two Phase 3 clinical trials of tezacaftor (VX-661) in combination with ivacaftor (Kalydeco®) showed positive results, Vertex Pharmaceuticals announced today.
Managing cystic fibrosis can feel overwhelming at times — but we’re here to help. Learn how to navigate life with CF.
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.
The U.S. Food and Drug Administration (FDA) announced that it has approved Ultresa™ delayed-release capsules, a pancreatic enzyme replacement therapy. Ultresa (pancrelipase) is manufactured by Aptalis Pharma.