Vertex Pharmaceuticals will present data on the long-term use of their cystic fibrosis (CF) treatments at the 43rd European Cystic Fibrosis Digital Conference, and then again at the 2020 North American Cystic Fibrosis Virtual Conference. The treatments being presented are TRIKAFTA, KALYDECO, and ORKAMBI.
About Cystic Fibrosis
Cystic fibrosis is a genetic disorder that is characterized by progressive damage to the respiratory and digestive systems. Those with cystic fibrosis do not have the slippery mucus that is normally found in the lungs. Instead they have thick and sticky mucus which builds up in their system. This buildup causes clogs in the airways, which then traps bacteria and causes breathing problems, infections, lung damage, and respiratory failure. It can also block digestive enzymes, which makes it difficult to absorb nutrients. Cystic fibrosis is a recessive disorder, meaning that the mutated gene must be passed down by both parents. The gene responsible for this condition affects the protein that regulates salt movement. The mutation in the gene varies in severity as well.
Symptoms of cystic fibrosis affect the respiratory and digestive systems. They include persistent coughing and wheezing, shortness of breath, difficulty exercising, frequent lung infections, stuffy noses, trouble with gaining weight, constipation, male infertility, salty-tasting skin, and exercise intolerance.
What’s Being Presented
One of the major things being presented is data from the open-label extension study of TRIKAFTA. This trial demonstrated a similar safety and efficacy profile as earlier studies of the treatment, which were conducted in cystic fibrosis patients over the age of 12 who have specific genotypes. There were improvements across all endpoints as well, including sweat chloride, body mass index, score on the Cystic Fibrosis Questionnaire Revised (CFQ-R) respiratory domain, and percent predicted forced expiratory volume in one second.
In addition, Vertex will put on six presentations in regards to their three treatments: TRIKAFTA, KALYDECO, and ORKAMBI, all of which are CFTR modulators. Not only will Vertex be presenting data, but they also published four abstracts on the medications in the Journal of Cystic Fibrosis.
Find out more about these treatments and presentations here.
