People 12 years and older are now approved by the FDA to take Trikafata, a new groundbreaking drug that targets the genetic root of Cystic Fibrosis.
What We Know:
- Cystic Fibrosis is a disease that causes thick mucus to build up in the lungs and other organs. It causes difficulty in breathing, smelly bowel movements, and salty-tasting skin. It’s caused by a defective protein that results from mutations in the CFTR gene. Trikafta merges three drugs to target the defective protein. This new drug will help these people’s quality of life.
- The drug is expected to help nearly 90% of patients with this disease. FDA Commissioner Ned Sharpless went on to say “Today’s landmark approval is a testament to [our] efforts, making a novel treatment available to most cystic fibrosis patients, including adolescents, who previously had no options and giving others in the cystic fibrosis community access to an additional effective therapy,” according to Considerable.
- Francis Collins discovered the gene defect in 1989. He’s excited about what this drug can do for future patients. “Thirty years along, with many bumps along the road and so many people waiting and hoping that something like this would happen — and here we are.”
Doctors are so encouraged by this drug’s potential that they are telling patients who rarely considered going to college that they should plan for retirement. Most patients before this lived only till their late 30’s.