According to a story from Medical Xpress, a recently published French study evaluating the impact of the combination drug lumacaftor-ivacaftor (marketed as Orkambi) concluded that the drug had the potential to provide multiple benefits for cystic fibrosis patients; however, not all patients were able to enjoy them. This is because the study found that around one in every five patients couldn’t tolerate the treatment.
About Cystic Fibrosis (CF)
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.
Study Results
Drug tolerance refers to a patient’s ability to take a drug without having to halt treatment as a result of serious adverse events or complications. The researchers found that at a year of follow-up, almost one in five cystic fibrosis patients using Orkambi had stopped treatment, typically because of adverse breathing events. The authors noted that this significant rate of halting treatment was higher than reported during the drug’s clinical trials. The study included a total of 847 patients (both adults and adolescents) that were treated at 47 different trial sites in France.
Despite these discouraging findings, Orkambi did provide some benefits for many patients, such as an increase of an average of 3.67 percent in forced expiratory volume (FEV1), a measure of lung function. The drug also reduced a patient’s need for intravenous antibiotics by an average of 35 percent and improved healthy weight gain.
Still, the 18.2 percent dropout rate was far higher than the less than five percent rate reported in trials, suggesting that participation criteria favored the impact of the drug in a way that is not being replicated by real-world data.
“Our study found that the benefits and risks of new therapies cannot be extrapolated to patients who were excluded from clinical trials.” – Dr. Pierre-Régis Burgel
Read the original study here.
