Recently, the US Food and Drug Administration (FDA) approved the drug ivacaftor, also known by the name Kalydeco for the treatment of a rare form of cystic fibrosis. This drug has now been tested for its efficacy by professionals at the University of Alabama at Birmingham.
This medication has been approved for individuals aged 6 and above who possess a minimum of 1 copy of the G551D mutation in the cystic fibrosis transmembrane conductance regulator gene (CFTR). The team conducted 4 studies for evaluating the new drug in patients with the G551D mutation.
Steven Rowe, M.D., MSPH, associate professor in the Division of Pulmonary, Allergy and Critical Care Medicine at the University of Alabama at Birmingham School of Medicine, commented, “Today was a big day for the CF community. I believe this new therapy will substantially improve the lives of CF patients with the G551D mutation, and we hope that with appropriate testing it will also soon be shown to help patients with other CFTR mutations as well.”
As per the outcomes, subjects who consumed Kalydeco seemed to encounter vast and consistent improvements with respect to lung function and other diseases. Conditions such as weight gain, and CF respiratory complications appeared to be alleviated too. Moreover, the overall quality of life seemingly improved to an extent, as compared to those who were treated with placebo.
Some of the side-effects associated with the drug were inclusive of diarrhea, common cold, upper respiratory tract infections, headache and stomach pain. The degree of these health outcomes ranged from mild to moderate.