Long-awaited cystic fibrosis drug could turn deadly disease into a manageable condition

Data unveiled Thursday revealed that a new cystic fibrosis therapy dramatically improved patients' lung function and showed clear signs of targeting the genetic root of the disease, instead of just alleviating symptoms.

Data unveiled Thursday revealed that a new cystic fibrosis therapy dramatically improved patients' lung function and showed clear signs of targeting the genetic root of the disease, instead of just alleviating symptoms. FDA last week approved the three-drug combination elexacaftor, tezacaftor, ivacaftor; ivacaftor (Trikafta—Vertex) 5 months ahead of the agency's deadline. The drug could benefit 90% of patients with the disease, a major advance over previous drugs that worked in a tiny fraction of the people with the disease or had more modest effects. The data, being unveiled Thursday at a national conference in Tennessee and simultaneously published in two leading medical journals, was so persuasive that FDA last week approved the three-drug combination, called Trikafta—5 months ahead of the agency's deadline. "I'm overjoyed," said NIH Director Francis Collins, MD, who was part of one of the teams that in 1989 discovered the gene defect that causes cystic fibrosis. "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." The drug is the product of decades of incremental scientific work that began with research in academic laboratories and was pushed forward and funded by patient advocates through an unusual "venture philanthropy" model now being emulated by other patient groups.