Cystic fibrosis is a life-threatening, genetic disease that affects patients’ ability to breathe and causes persistent lung infections. There is currently no cure for cystic fibrosis, but there are treatments designed to help patients maintain their health and have longer, more fulfilling lives. There are cystic fibrosis medications approved for patients with the disease, but treatment plans are unique and tailored to each patient’s specific health characteristics and needs. The range of cystic fibrosis medications includes CFTR modulators, enzyme supplements, mucolytics, antibiotics, and vitamins.
CFTR Modulators for Cystic Fibrosis
The Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) is a protein which regulates the proper flow of chloride and sodium (salt) in and out of cells lining the lungs and other organs. Patients who suffer from cystic fibrosis have mutations in the gene that provoke a malfunction of the CFTR protein, which is why patients usually suffer from a buildup of thick mucus, persistent lung infections, pancreatic damage, and complications in other parts of the body. CFTR modulators are the main course of treatment for cystic fibrosis and include:
In March, the U.K. National Institute for Health and Care Excellence (NICE) issued a draft guidance against recommending Orkambi (lumacaftor-ivacaftor) for treating cystic fibrosis (CF) since its cost is too high for the low benefits it brings.