Vertex receives FDA approval for expanded use of cystic fibrosis therapy

 

• Vertex Pharmaceuticals (NASDAQ:VRTX) announced that it received the FDA signoff for the expanded use of three of its cystic fibrosis medications.
• TRIKAFTA, previously approved for people with at least one F508del mutation is now approved for 177 additional mutations; authorized for 127 additional mutations, SYMDEKO is now approved for a total of 154 SYMDEKO-responsive mutations; KALYDECO is now eligible for use in a total of 97 KALYDECO-responsive mutations after getting approval for an additional 59 mutations.
• TRIKAFTA is indicated for people with cystic fibrosis aged 12 years and older, while SYMDEKO and KALYDECO are approved for people with CF ages six years and older and age four months and older, respectively
• Cystic fibrosis, due to mutations in the cystic fibrosis transmembrane conductance regulator gene, is a rare, life-shortening genetic disease affecting approximately 75,000 people worldwide.
• With the vast majority of CF patients having at least one F508del mutation, the approval for expanded use will benefit more than 600 people with CF, additionally.
• https://seekingalpha.com/news/3646333-vertex-receives-fda-approval-for-expanded-use-of-cystic-fibrosis-therapy