When the U.S. Food and Drug Administration announced two years ago it had approved Trikafta (elexacaftor/tezacaftor/ivacaftor) for some cystic fibrosis (CF) patients 12 and older, I carried what I considered to be a healthy amount of skepticism about the hype. Articles like this one from The Washington Post…

A new agreement will give people in Scotland with cystic fibrosis access to the CF combination treatments Orkambi (lumacaftor/ivacaftor) and Symkevi (tezacaftor/ivacaftor) through the country’s health system, according to Vertex Pharmaceuticals, the company that sells both medicines. The announcement follows a prior rejection by the …

Cystic fibrosis (CF) patients in Australia age 12 and older, who have two copies of the F508del mutation in the CFTR gene (a copy inherited from each parent), now can be treated with Vertex Pharmaceuticals’ Symdeko (tezacaftor/ivacaftor and ivacaftor). The treatment also was approved for any mutation in the CFTR gene that responds to Symdeko based on lab…

Vertex Pharmaceuticals’ Symkevi (tezacaftor/ivacaftor) is one step closer to being approved in the European Union as a treatment for cystic fibrosis (CF) patients ages 12 or older who have certain CFTR genetic mutations. The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency…

Vertex Pharmaceuticals, a clinical-stage biopharmaceutical company, recently announced that two Phase 3 studies evaluating its tezacaftor/ivacaftor combination therapy have shown statistically significant and clinically meaningful improvements in lung function in patients with certain mutations associated with cystic fibrosis (CF). The first study, called EVOLVE (NCT02347657), was conducted to…

Vertex Pharmaceuticals is continuing to develop treatments for all people with cystic fibrosis (CF), with clinical trials exploring combinations of its potentiator and corrector therapies. At the same time, efforts to expand the use of already-approved treatments are ongoing. In addition, the company recently completed the purchase of Concert’s…