Vertex gets FDA approval for Kalydeco sNDA

Treatment will be carried out in CF patients aged six and older who have one of eight additional mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

In January 2012, Kalydeco was approved for people with CF aged six and older who have at least one copy of the G551D mutation.

With the approval of the sNDA, Kalydeco is now approved for use in CF patients with nine mutations including G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P and G1349D.

In the US, about 150 people aged six and older have one of the additional eight mutations for which Kalydeco is now approved.

Vertex senior vice president and co-chief medical officer Robert Kauffman said the company believes that Kalydeco has the potential to help more people with CF, and the approval is an important step toward that goal.

"As we progress over the coming year, we look forward to data from multiple other ongoing studies that are designed to evaluate whether additional people with CF may benefit from kalydeco," Kauffman said.

The drug was also granted Breakthrough Therapy designation by the US FDA in late 2012.

The sNDA approval is based on data from a Phase III, two-part, randomized, double-blind, placebo-controlled, cross-over study of 39 people with CF who had one of the following mutations: G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, G1349D or G970R.

CF is caused by defective or missing CFTR proteins that results in poor flow of salt and water into and out of the cell in a number of organs, including the lungs.

Image: Vertex’s Kalydeco is intended for the treatment of CF patients aged six and older who have one of eight additional mutations in the CFTR gene. Photo: courtesy of Vichaya Kiatying-Angsulee/ freedigitalphotos.net