Vertex Announces Positive Results from Phase 3 Study of Kalydeco in Select CF Patient Groups 

July 30, 2013

Vertex Pharmaceuticals Inc. yesterday announced initial results from a Phase 3 clinical trial of Kalydeco™ (ivacaftor) in people with certain mutations of the cystic fibrosis gene. Study participants who have what are known as gating mutations who took the drug showed statistically significant improvements in lung function and weight gain, compared with those who received a placebo.

Kalydeco, the first drug to address the underlying cause of CF, was approved by the U.S. Food and Drug Administration (FDA) in 2012 to treat people with the G551D mutation ages 6 and older.

The current trial tested Kalydeco in people with CF ages 6 and older with non-G551D gating mutations. The trial is one of several ongoing studies of Kalydeco in CF patient groups that were not evaluated in earlier clinical trials of the drug.

“We welcome these promising first results from the ongoing trials to expand the use of Kalydeco to greater numbers of people with CF,” said Robert J. Beall, Ph.D., president and CEO of the Cystic Fibrosis Foundation. “If approved for these patients, Kalydeco would become available to about 400 more people with gating mutations of CF, in addition to the hundreds already benefiting from the drug.”

G551D is the most common gating mutation, found in about 4 percent of people with CF in the United States. The remaining gating mutations are found in about 1 percent of those living with the disease.

In gating mutations like G551D, the defective protein in CF moves to its proper place at the surface of the cell but does not function correctly. Instead, it acts like a locked gate. Kalydeco is designed to unlock that gate, allowing the proper flow of salt and water in and out of the cells lining the airways and other organs. 

Vertex said it plans to submit a new drug application to the FDA in the second half of 2013 for approval of Kalydeco to treat people ages 6 and older with at least one copy of a non-G551D gating mutation of CF.

Kalydeco is also being studied in combination with another potential therapy, VX-809, in two large, international Phase 3 trials of people with two copies of the most common mutation of CF, Delta F508.

“We are also very encouraged that, around the world, people with two copies of the most common CF mutation are now enrolling in the Phase 3 Kalydeco and
VX-809 combination trials,” said Beall. “These are potentially among the most important studies in the history of the disease.”

Read the press release from Vertex.