U.S. approves breakthrough cystic fibrosis drug

Female scientist holding up test tube (© Gregory Bull/AP Images)
Vertex Pharmaceuticals Incorporated spends billions developing treatments for rare diseases. A scientist works in the company’s San Diego laboratory in 2015. (© Gregory Bull/AP Images)

U.S. innovators have developed a new drug for treating cystic fibrosis that will dramatically expand treatment options for patients suffering from the life-threatening disease.

The new treatment will help 90 percent of cystic fibrosis patients, including many who previously lacked treatment options, the U.S. Food and Drug Administration (FDA) said in October when it approved Vertex Pharmaceuticals Incorporated’s treatment, called Trikafta.

Vertex Pharmaceuticals, a biotechnology firm headquartered in Boston, combined three drugs to make Trikafta, which targets a defective protein that causes cystic fibrosis, a hereditary disease that affects the lungs and digestive system.

Trikafta’s approval “marks a tremendous breakthrough” for patients with the disease, said Preston W. Campbell III, president of the Cystic Fibrosis Foundation.

While other treatments exist, former FDA Commissioner Ned Sharpless said Trikafta is the first treatment approved for adolescents with the most common form of the disease and will provide additional treatment options for others with the disease.