According to a story from BioSpace, the US Food and Drug Administration (FDA), has officially approved the drug Trikafta as a treatment for cystic fibrosis, a rare disease that causes a progressive decline in lung function. The drug is approved for patients that are at least twelve years old. The drug consists of three active components (tezacaftor, elexacaftor, and ivacaftor) and is expected to be effective for about 90 percent of the patient population.
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.
Improving Patient Treatment
The approval of Trikafta means that are larger number of cystic fibrosis patients now have the potential to see benefit from a single treatment than ever before. The drug’s three components are designed to act on the most common of the CFTR mutations, which is known as F508del. Around 27,000 patients in the US are expected to carry such a mutation.
The effectiveness of the drug was evaluated in two clinical trials. The first study involved 403 patients and the treatment was able to improve forced expiratory volume (FEV), a critical measure of lung function, by a mean of 13.8 percent when compared to placebo. The second trial involved 107 patients and the treatment was able to improve FEV by a mean of 10 percent when compared to a two-part combination of tezacaftor and ivacaftor. The treatment was also able to provide improvements in the rate of pulmonary exacerbations (incidents of disease progression) and sweat chlorides.
