A new cystic fibrosis drug is the first of its kind. According to the Associated Press, Vertex Pharmaceuticals’ Kalydeco is the first cystic fibrosis drug to combat the root cause of the inherited illness that causes early death.
Kalydeco treats a rare form of the disease caused by a defective protein that prevents a patient’s cells from absorbing and excreting water. The pill is taken twice a day and was shown to improve lung function and other symptoms of cystic fibrosis during clinical trials.
This form of cystic fibrosis affects only about four percent of people with the disease, or just 1,200 people in the U.S. Still, researchers are celebrating the drug’s approval after more than two decades of work.
“Even though this drug isn’t for the majority of people, it proves that you can look at the mistake in the genes and design a drug in a rational way that will fix it,” said Dr. Drucy Borowitz of the State University of New York.
Kalydeco successfully lowered the rate of infections among cystic fibrosis patients, helping them to gain weight, increase their lung strength and rid themselves of mucus. The drug was approved for patients six years old and up.
Vertex hopes to develop similar medication for patients as young as two years old, the AP said. Using the drug earlier may help to prevent lung damage later in life.
According to the Cystic Fibrosis Foundation, the disease causes mucus buildup in the lungs and other organs that leads to infections and digestive problems. The average life expectancy for someone with cystic fibrosis is 37 years.
