FDA approves new breakthrough therapy for cystic fibrosis
The U.S. Food and Drug Administration today approved Trikafta (elexacaftor/ivacaftor/tezacaftor), the first triple combination therapy available to treat patients with the most common...
FDA Approves Trikafta for 94 Additional Rare CFTR Mutations
Dec 20, 2024 · The U.S. Food and Drug Administration (FDA) today approved the expansion of Trikafta (elexacaftor/tezacaftor/ivacaftor) to people with cystic fibrosis ages 2 and older who have at least one of 94 rare mutations in the cystic fibrosis …
Vertex Highlights First Oral Presentation of Phase 3 Clinical Data of ...
Sep 26, 2024 · Vertex will present new data on TRIKAFTA ® from long-term (96 week and 192 week) studies in patients ages 2-11 years old that reinforce the sustained benefit seen in studies in older people with CF. Specifically, that early treatment with TRIKAFTA ® is associated with sustained improvements in lung function.
FDA Approves Expanded Use for Trikafta in Cystic Fibrosis
Dec 20, 2024 · Trikafta's expanded approval includes patients aged 2+ with F508del or responsive mutations, benefiting 300 more US patients. Trikafta improves CFTR protein function, enhancing lung function and reducing cystic fibrosis symptoms. Clinical trials showed significant improvements in ppFEV1, sweat chloride, and other health markers with Trikafta.
The Cystic-Fibrosis Breakthrough That Changed Everything
Mar 7, 2024 · Then, in the fall of 2019, a new triple combination of drugs began making its way into the hands of people with the genetic disease. Trikafta corrects the misshapen protein that causes cystic...
FDA Approval Increases Eligibility for Cystic Fibrosis Medication Trikafta
3 days ago · Findings showed Trikafta significantly improved ppFEV 1 at week 24 compared with placebo (treatment difference, 9.2 [95% CI, 7.2-11.3]; ... News release. Vertex Pharmaceuticals. December 20, 2024.
FDA Approves Trikafta for Children Ages 2 Through 5 Years With …
Apr 26, 2023 · The U.S. Food and Drug Administration (FDA) has approved the use of Trikafta ® (elexacaftor/tezacaftor/ivacaftor) for children with cystic fibrosis ages 2 through 5 years who have at least one copy of the F508del mutation or certain mutations that are responsive to Trikafta based on lab data.
Vertex' Trikafta Gets Expanded FDA Approval For Cystic Fibrosis ...
Dec 20, 2024 · With this approval, 94 additional non-F508del CFTR mutations have been added to the Trikafta label, and approximately 300 additional people with CF in the U.S. are now eligible for a medicine to ...
Vertex wins Trikafta expansion and approval for 'vanza triple' …
Dec 23, 2024 · That’s higher than the twice-daily Trikafta wholesale acquisition cost of $346,048 per year. Minter said William Blair “conservatively” modeled greater than $11.5 billion in peak sales for ...
Vertex Announces U.S. FDA Approval for TRIKAFTA (elexacaftor
Dec 23, 2024 · TRIKAFTA should not be used in patients with severe hepatic impairment (Child-Pugh Class C). TRIKAFTA is not recommended in patients with moderate hepatic impairment (Child-Pugh Class B). ... BioSpace is the digital hub for life science news and jobs. We provide essential insights, opportunities and tools to connect innovative organizations and ...