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.
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.
Update: On February 3, 2017, the Cystic Fibrosis Foundation divested its remaining ownership stake in CF Services Inc., a specialty pharmacy. The pharmacy is now fully owned by Walgreens.
Aim is to Provide Cystic Fibrosis Patients Enhanced Access to Medications and Expanded Pharmacy Services
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.