Combination of Two Drugs helps Patients suffering from Common Cystic Fibrosis
Researchers announced on Sunday that two recent trials of combination of two drugs, lumacaftor and ivacaftor, has shown promising results towards improving health of patients suffering from most common form of cystic fibrosis, a genetic disorder that mostly affects the lungs.
According to the researchers, results of the clinical trials have shown that people suffering from the genetic disorder who were treated with the two drugs saw improvement in their ability to breathe. In addition, the patients noticed that they had fewer lung infections than the patients who took a placebo, as per the results of the trials that have been published in the New England Journal of Medicine.
The clinical trials included more than 1,100 patients aged 12 and older. During the trials, the patients were treated for about six months. Susanna McColley, a professor of pediatrics at Northwestern University's Feinberg School of Medicine and author of the new study, said the results of the study will help about 15,000 people in the United States.
As per some reports, more than 70,000 people in Australia, Europe and North America develop cystic fibrosis, which is usually caused by genetic mutations. People develop the disease when they have about two copies of the mutation named ‘F508del mutation’. About 50% of all cystic fibrosis patients have two copies of the mutation.
About three years ago, the drug Ivacaftor became the first medicine of its type to be approved by the United States Food and Drug Administration (FDA). The drug showed in a number of studies that it could help in improving the health of patients suffering from a more rare genetic mutation for CF. The other drug in the combination, lumacaftor, is yet to be approved.
According to an announcement by Vertex, an FDA advisory panel voted 12-1 to approve the two-drug combination.