FDA Approves New Cystic Fibrosis Drug
FDA Approves New Cystic Fibrosis Drug

FDA Approves New Cystic Fibrosis Drug

The treatment, Trikafta, increases lung function in most patients with the disease—but comes with a hefty price tag.

Oct 24, 2019
Shawna Williams

ABOVE: BUSINESS WIRE

The Food and Drug Administration announced on Monday (October 21)—months ahead of its expected decision date—that it has approved a new treatment for cystic fibrosis. Trikafta, made by Vertex Pharmaceuticals, is, as its name suggests, a combination of three drugs. It works for 90 percent of patients with cystic fibrosis, and will be priced at $311,000 per year. 

“Now, I know that my son’s lungs will function as well as mine. That’s just unbelievable,” Margot Cleveland, whose 10-year-old son has cystic fibrosis, tells STAT of the approval. 

Cystic fibrosis is a rare genetic disorder that affects about 30,000 people in the United States. It can be caused by a variety of mutations in the gene CFTR, which codes for a protein involved in moving salt in and out of cells. People with the disease produce unusually sticky mucus that plugs up the lungs and other organs. Trikafta works for patients who have the most common disease-causing CFTR mutation, known as F508del, by bolstering the function of the defective salt-moving protein.

Trikafta is a “transformative medication which is going to significantly improve outcomes for people with cystic fibrosis,” Mike Boyle, senior vice president of therapeutics development at the Cystic Fibrosis Foundation, which funded early research toward the drug, tells STAT.

The outlet notes that Trikafta was developed unusually quickly, in part because Vertex opted to test several different three-drug combinations simultaneously (the company already had one-drug and two-drug cystic fibrosis treatments on the market). In trials, the drug increased the amount of air that patients could exhale in one second by 14 percent.