TRIKAFTA®

Health Canada Grants Marketing Authorization for TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) in People Ages 12 and Older Who Have at Least One F508del Mutation

Retrieved on: 
Friday, June 18, 2021
Research, Genetics, Clinical trials, Other Health, Biotechnology, General Health, Pharmaceutical, Health, Science, Other Science, Cystic fibrosis, Drugs, Health sciences, Orphan drugs, Health care, Breakthrough therapy, Combination drugs, Cystic fibrosis transmembrane conductance regulator, Elexacaftor/tezacaftor/ivacaftor, Reshma Kewalramani, Vertex, Vertex Pharmaceuticals, TRIKAFTA®, Cystic Fibrosis, Vertex, TRIKAFTA®, CYSTIC FIBROSIS, VERTEX

With this approval, for the first time, approximately 1,100 eligible patients with CF ages 12 years and older who have at least one F508del mutation have a medicine that targets the underlying cause of their CF.

Key Points: 
  • With this approval, for the first time, approximately 1,100 eligible patients with CF ages 12 years and older who have at least one F508del mutation have a medicine that targets the underlying cause of their CF.
  • The approval of TRIKAFTA marks a significant milestone for Canadians with CF, their families and Vertex, said Reshma Kewalramani, M.D., Chief Executive Officer and President, Vertex.
  • TRIKAFTA is designed to increase the quantity and function of the F508del-CFTR protein at the cell surface.
  • Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

Vertex Announces Positive Phase 3 Study for TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) in Children Ages 6-11 Years With Cystic Fibrosis to Support Submissions for Global Regulatory Approvals

Retrieved on: 
Thursday, September 10, 2020
Research, FDA, Genetics, Clinical trials, Biotechnology, Health, Pharmaceutical, Other Science, Science, Anatomy, Medical specialties, Organ systems, Cystic fibrosis, RTT, Pancreas disorders, Channelopathies, Breakthrough therapy, Elexacaftor/tezacaftor/ivacaftor, Gastrointestinal tract, Cystic Fibrosis, TRIKAFTA®

We are looking forward to filing an sNDA in the coming months and bringing TRIKAFTA to younger people with CF.

Key Points: 
  • We are looking forward to filing an sNDA in the coming months and bringing TRIKAFTA to younger people with CF.
  • Based on the results, Vertex will submit an sNDA to the U.S. FDA in the fourth quarter of 2020, with additional global regulatory submissions to follow.
  • Cystic Fibrosis (CF) is a rare, life-shortening genetic disease affecting approximately 75,000 people worldwide.
  • CF is a progressive, multi-system disease that affects the lungs, liver, GI tract, sinuses, sweat glands, pancreas and reproductive tract.

Positive Phase 3 Study Results for TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) in People Ages 12 and Older With Cystic Fibrosis Who Have One Copy of the F508del Mutation and One Gating or Residual Function Mutation

Retrieved on: 
Monday, July 20, 2020
Health, FDA, Genetics, Other Health, Clinical trials, Pharmaceutical, Biotechnology, Drugs, Chemical compounds, Orphan drugs, Chemistry, Breakthrough therapy, Ivacaftor, Phenols, Elexacaftor/tezacaftor/ivacaftor, Tezacaftor, Vertex Pharmaceuticals, the 445-104 Study, Cystic Fibrosis, TRIKAFTA®

The regimen was generally well-tolerated, and safety data were consistent with those observed in previous Phase 3 studies with TRIKAFTA.

Key Points: 
  • The regimen was generally well-tolerated, and safety data were consistent with those observed in previous Phase 3 studies with TRIKAFTA.
  • The study is a post-marketing commitment in the U.S. and the results will be submitted to the U.S. Food and Drug Administration.
  • Full study results will be submitted for presentation at a future medical meeting and/or publication.
  • Following the run-in, patients were randomized to receive TRIKAFTA or to remain on their prior regimen of ivacaftor or tezacaftor/ivacaftor for 8 weeks.