FDA Approval Increases Eligibility for Cystic Fibrosis Medication Trikafta
1 day 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 94 Additional Rare CFTR Mutations
Dec 20, 2024 · Trikafta was first approved in 2019 for people with CF ages 12 and older with at least one copy of the F508del mutation. Since then, the modulator has been expanded to people with CF ages 2 and older who have at least one copy of 178 specified mutations. With today’s announcement, Trikafta is approved to treat the following additional mutations:
Vertex Announces U.S. FDA Approval for TRIKAFTA …
Dec 20, 2024 · TRIKAFTA is not recommended in patients with moderate hepatic impairment (Child-Pugh Class B). If used, use with caution at a reduced dosage and monitor patients closely. WARNINGS AND PRECAUTIONS. Drug-Induced Liver Injury and Liver Failure. TRIKAFTA can cause serious and potentially fatal drug-induced liver injury.
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 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.
Vertex Wins FDA Approval for New Cystic Fibrosis Option but
5 days ago · The new combination, dubbed Alyftrek, is designed to improve on the Trikafta, a product that generated sales of $8.9 billion in 2023. It’s welcome good news for Vertex following last week’s subpar clinical results for its non-opioid …
Vertex wins Trikafta expansion and approval for 'vanza triple' …
5 days ago · 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 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 Trikafta for Children Ages 2 Through 5 Years With …
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 ...