A 3-drug combination can treat the cystic fibrosis (CF) effectively, as per the results of the phase-3 trials which were completed and already approved by the FDA, USA.
Trial determined that a three-drug combination improved lung function and reduced symptoms in cystic fibrosis (CF) patients who have a single copy of the most common genetic mutation for the disease.
CF is a chronic, progressive, and frequently fatal genetic disease that affects the respiratory and digestive systems in children and young adults. The sweat glands and the reproductive system are also usually involved. Individuals with CF have a shortened lifespan.
Although there are over a thousand different disease-causing mutations, nearly 90 percent of people with cystic fibrosis have at least one copy of the most common mutation, the Phe508del CFTR allele, according to the researchers.
An estimated 80,000 people worldwide are affected by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) protein, she said. People inherit a gene from each parent that encodes the CFTR protein.
In the clinical trial conducted at 115 sites in 13 countries from June 2018 to April 2019, 403 patients of ages 12 and older were randomized to receive either elexacaftor-tezacaftor-ivacaftor combined therapy or a placebo. The trial was co-sponsored by Vertex Pharmaceuticals.