The therapy — a triple combo based on elexacaftor, tezacaftor, and ivacaftor, which is being developed by Vertex Pharmaceuticals — was approved on Oct. 21 for people with cystic fibrosis (CF) who are 12 and older, and who have at least one copy of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. This is the most common CF-causing mutation; it is estimated to be present in nine out of 10 people with CF.
Trikafta works by improving the ability of the protein encoded by the mutated gene to function properly.
“Today marks a tremendous breakthrough and exciting news for people with cystic fibrosis,” Preston W. Campbell, III, MD, president and CEO of the CFF, said in a news release. “This milestone is the result of an extraordinary community working together against great odds, and we are overjoyed that this will mean more people will have effective treatments for their disease.”
In clinical trials (NCT03525444 and NCT03525548), Trikafta increased lung function (measured by ppFEV1, the percent predicted forced expiratory volume in one second, which is a widely used measure of lung function) by an average of 13.8 percentage points in people with one F508del mutation, compared to treatment with a placebo. In people with two F508del mutations, Trikafta increased lung function by 10 percentage points compared to those treated with Symdeko (tezacaftor/ivacaftor; an approved therapy also marketed by Vertex).
In terms of safety, the triple combo was generally well-tolerated in both studies.
Additional clinical trials to test Trikafta in children ages 6 to 11 are currently ongoing, with results expected in 2020.
According to Vertex, Trikafta has the potential to treat up to 90% of all CF patients. Nonetheless, CFF emphasized that more work is needed to find effective treatments for all patients.
“As we celebrate today, we will not lose sight of the many individuals in our community who are still waiting for a breakthrough that will treat their mutations,” said Michael P. Boyle, MD, senior vice president of therapeutics development at CFF.
“We will not be done until every person with CF has a treatment for the underlying cause of their disease and, one day, a cure,” Boyle said.