According to a story from Xconomy, the drug developer Vertex Pharmaceuticals recently announced that a three drug combination treatment for the rare lung disorder cystic fibrosis has performed well in Phase 3 clinical trials. The results could indicate another advancement in treatment options for this disease; the combination has the potential to effectively treat up to 90 percent of patients.
About Cystic Fibrosis
Cystic fibrosis is a type of genetic disorder which can have impacts throughout the body, but it is most characterized by the build up of abnormally thick, sticky mucus in the lungs. This mucus becomes a fertile breeding ground and habitat for potentially infectious bacteria. Many patients must take antibiotics for much of their lives. This disorder is caused by mutations of the CFTR gene. Symptoms of cystic fibrosis include progressive decline in lung function, lung and sinus infections, coughing up mucus, fatty stool, poor growth, infertility in males, clubbed digits, and digestive problems. Treatment includes antibiotics and medications or procedures intended to maintain lung function. Lung transplant is an option when lung function declines severely. Life expectancy ranges into the 40s and 50s with good care. To learn more about cystic fibrosis, click here.
Vertex in the Lead
Vertex has already established itself as a cutting edge developer when it comes to treating cystic fibrosis. The combination therapy consists of the two active ingredients in Symdeko, an already approved therapy from the company for cystic fibrosis. These two drugs are called ivacaftor and tezacaftor. In the study, these two compounds were combined with an investigational new therapy from the company known as VX-659.
There is another triple drug combination that the company has been testing alongside this one; once the trials for each are completed, Vertex intends to file the most effective one for approval from the US Food and Drug Administration (FDA) by the middle of next year.
In the study, the VX-659-tezacaftor-ivacaftor combination was able to produce clinically meaningful improvements in lung functionality after a month of treatment. This increase was 10 percent in one study of 111 patients and 14 percent in a study of 382 patients.
Vertex is also behind the cystic fibrosis drug Orkambi, and with both this drug and Symdeko its therapies can treat about 40 percent of patients currently. A three part combination appears to be the key for treating a greater percentage of patients.