According to a story from Market Watch, the biotechnology company Vertex Pharmaceuticals, Inc. has announced recently that the US Food and Drug Administration (FDA) has accepted the company’s New Drug Application (NDA). This application is for the investigational drug VX-445, also known as elexacaftor. This drug combines three different active ingredients to treat cystic fibrosis, a rare disorder that mostly affects lung function.
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.
About Elexacaftor and the NDA
The FDA also granted Priority Review in regards to the NDA, setting a target date for review of the drug for March of next year. Priority Review means that the drug will be reviewed in a shorter time frame that it would have under normal conditions. The submission of the NDA for elexacaftor is supported by encouraging results from two phase 3 clinical trials. The drug was able to cause significant improvements in lung functionality and also met all secondary endpoints. The drug is intended for cystic fibrosis patients that carry either a F508del mutation and a minimal function mutation or two F508del mutations.
While there are some drugs available for cystic fibrosis, they are limited in effectiveness and there is still a significant need for more impactful treatment options. Hopefully, elexacaftor will become a new breakthrough therapy for this group of rare disease patients.