The New England Journal of Medicine

Tezacaftor–Ivacaftor in Residual-Function Heterozygotes with Cystic Fibrosis

Cystic fibrosis is an autosomal recessive disease caused by mutations in the CFTR gene that lead to progressive respiratory decline. Some mutant CFTR proteins show residual function and respond to the ...
The New England Journal of Medicine

Lumacaftor–Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del CFTR

Cystic fibrosis is a life-limiting disease that is caused by defective or deficient cystic fibrosis transmembrane conductance regulator (CFTR) protein activity. Phe508del is the most common CFTR ...
Medical Xpress

Lumacaftor, ivacaftor linked to improved lung function in CF

(HealthDay)—For patients aged 6 to 11 years with cystic fibrosis homozygous for F508del-cystic fibrosis transmembrane conductance regulator (CFTR), lumacaftor and ivacaftor treatment is associated ...
The American Journal of Managed Care

Tezacaftor, Ivacaftor Combination Generally Safe, Well Tolerated in Children With CF Under Age 12

Data from a phase 3 trial are supporting the use of tezacaftor in combination with ivacaftor for the treatment of cystic fibrosis (CF) in children aged 6 to 11 years, finding that the treatment ...
National Institute for Health and Care Excellence

Ivacaftor–tezacaftor–elexacaftor, tezacaftor–ivacaftor and lumacaftor–ivacaftor for treating cystic fibrosis

There is a commercial access agreement for ivacaftor–tezacaftor–elexacaftor, tezacaftor–ivacaftor, and lumacaftor–ivacaftor. NHS organisations can get details on the Commercial Access and Pricing (CAP ...
FiercePharma

FDA Advisory Committee Recommends Approval of KALYDECO® (ivacaftor) for Use in People with Cystic Fibrosis Ages 6 and Older Who Have the R117H Mutation

BOSTON--(BUSINESS WIRE)-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the U.S. Food and Drug Administration's Pulmonary Allergy Drugs Advisory Committee (PADAC) voted 13-2 ...
Nasdaq

ADDING MULTIMEDIA FDA Approves TRIKAFTA (elexacaftor/tezacaftor/ivacaftor and ivacaftor) to Treat the Underlying Cause of Cystic Fibrosis in People Ages 12 and Older Who Have ...

- For the first time, approximately 6,000 patients with one minimal function mutation and one F508del mutation have a medicine to treat the underlying cause of their disease– -12,000 people with one ...
The American Journal of Managed Care

Study Analyzes Benefits of Ivacaftor Treatment for Cystic Fibrosis

Patients with cystic fibrosis (CF) who were treated with ivacaftor have demonstrated favorable results for clinically important outcomes, including a lower prevalence of CF-related complications and ...
Medindia

Tezacaftor and Ivacaftor - Indications, Dosage, Side Effects and Precautions

Get detailed information on Tezacaftor and Ivacaftor, including pronunciation, uses, dosage guidelines, indications, and instructions on how and when to take it and when to avoid it. The updated ...
Health Affairs

Cystic Fibrosis And Ivacaftor Use: The Authors Reply

Almut Winterstein and Amir Sarayani have three concerns regarding our article (May 2018). Their first concern is mortality (“likely while hospitalized”). Of the 199 patients enrolled twelve months ...
Nasdaq

Phase 3 Results from Two Studies of TRIKAFTA (elexacaftor/tezacaftor/ivacaftor and ivacaftor) Triple Combination Treatment for Cystic Fibrosis Concurrently Published in The New ...

- Both studies met primary and all key secondary endpoints demonstrating significant improvements in lung function and other measures of the disease - - Results of both studies to be presented today ...