Trikafta is a new drug on the market that is a life-changer for many cystic fibrosis patients. According to the Genetics Home Reference, a consumer health website from the National Library of Medicine, cystic fibrosis is a devastating chronic disease that is caused by a mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. It is characterized by a buildup of thick, sticky mucus in the lungs, causing breathing problems and bacterial infections. People with cystic fibrosis often have low immunity, so even a cold can be life-threatening. The frequency of infections means that many people with the disease spend weeks in the hospital at a time, which can amount to millions of dollars spent on hospital care. GoodRx, a startup company that tracks prescription drug prices, explains that until now, cystic fibrosis was treated through drugs that only 6% of the population responded to, while the new drug can treat up to 90% of all patients. How does this groundbreaking drug work, and why is it such a game changer?
According to CenterWatch, a company that disseminates clinical trials information, Trikafta is a triple-modulator. This means that it fixes the genetic mutation that causes cystic fibrosis and restores normal function in the gene. Specifically, it increases the amount of CFTR protein delivered to the cell surface and opens the chlorine channel, allowing for the regulation of fluids at the cell surface. In trials comparing people with two chromosomal mutations, 10% of lung function was restored by the new drug, and people with only one chromosomal mutation had a 14% increase in lung function. The main difference between Trikafta and other drugs on the market is its ability to target only one copy of the F508del genetic mutation, which is the most common mutation found in patients.
Newsday explains that the drug currently costs $24,000 per year, which is covered partially by insurance. While this may seem radically expensive, compared to the millions spent in hospitals on patients recovering from infections, this is a better alternative for many people with cystic fibrosis. Trikafta promises to help cystic fibrosis patients stay out of the hospital and be healthier.