Posted by Michael Wonder on 21 Oct 2019
FDA approves new breakthrough therapy for cystic fibrosis
21 October 2019 - Treatment approved for approximately 90% of patients with cystic fibrosis, many of whom had no approved therapeutic options.
The U.S. FDA today approved Trikafta (elexacaftor/ivacaftor/tezacaftor), the first triple combination therapy available to treat patients with the most common cystic fibrosis mutation. Trikafta is approved for patients 12 years and older with cystic fibrosis who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, which is estimated to represent 90% of the cystic fibrosis population.
The efficacy of Trikafta in patients with cystic fibrosis aged 12 years and older was demonstrated in two trials. The first trial was a 24-week, randomised, double-blind, placebo-controlled trial in 403 patients who had an F508del mutation and a mutation on the second allele that results in either no CFTR protein or a CFTR protein that is not responsive to ivacaftor or tezacaftor/ivacaftor alone. The second trial was a four-week, randomised, double-blind, active-controlled trial in 107 patients who had two identical F508del mutations.
