The FDA has approved a supplemental New Drug Application (sNDA) for Kalydeco (ivacaftor) for people with cystic fibrosis ages 6 and older who have one of eight additional mutations in the CF transmembrane conductance regulator (CFTR) gene.
The FDA approval was announced by Vertex Pharmaceuticals Inc, the manufacturer of the drug, which was first approved in January 2012 for people with CF ages 6 and older who have at least one copy of the G551D mutation.
Tthe newest approval means Kalydeco can now be used to treat CF patients (age 6 and older) with the following nine mutations: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P and G1349D.
In the US, approximately 150 people ages 6 and older have one of these eight additional mutations, according to Vertex Pharm.
“We believe that Kalydeco has the potential to help more people with CF, and today’s approval is an important step toward that goal,” said Robert Kauffman, MD, PhD, senior vice president and co-chief medical officer at Vertex. “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.”
The sNDA approval is based on previously announced data from a Phase 3, 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.
The study showed statistically significant improvements in FEV1 for people in the overall study population who received ivacaftor, and the safety profile was similar to prior Phase 3 studies in people with the G551D mutation, according to Vertex Pharm.
The efficacy of Kalydeco could not be established to support US approval for patients with the G970R mutation.