Vertex Pharmaceuticals’ cystic fibrosis (CF) drug is now approved in the US for an expanded group of patients.
The company announced that the US Food and Drug Administration (FDA) has approved Kalydeco (ivacaftor) for use in children ages 2-5 with CF who have one of 10 mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. The drug was already approved for patients six years and older who have one of 10 mutations in the CFTR gene, including G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, S549R and R117H.
With today’s approval, Kalydeco will now be available for the approximately 300 children in the US ages 2 to 5 who have one of these 10 mutations, including 150 who have the R117H mutation and 150 who have one of the other nine mutations that result in a gating defect in the CFTR protein. In order to meet the needs of this age group, Vertex created a new weight-based oral granule formulation of the drug that can be mixed in soft foods or liquids.
The expanded approval is based on previously announced results of a Phase III 24-week study that was designed to evaluate the safety and pharmacokinetics of weight-based dosing of Kalydeco in children ages 2 to 5.
CF is a rare, life-threatening genetic disease which is caused by a defective or missing CFTR protein resulting from mutations in the CFTR gene. Children must inherit two defective CFTR genes, one from each parent, to have CF. There are more than 1,900 known mutations in the CFTR gene. Some of these mutations lead to CF by creating defective or too few CFTR protein at the cell surface. The defective function or absence of CFTR proteins in CF patients results in poor flow of salt and water into and out of the cell in various organs. In the lungs, this leads to the buildup of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage. Today, the median predicted age of survival for a person with CF is between 34 and 47 years, however the median age of death remains in the mid-20s.
“Children with cystic fibrosis can begin to experience meaningful lung function decline and struggle to gain weight at a very young age, underscoring the importance of starting treatment early in life,” said Jeffrey Chodakewitz, MD, Executive Vice President and Chief Medical Officer at Vertex. “With today’s approval, children as young as two years of age now have a medicine to treat the underlying cause of their CF, bringing us one step closer to our goal of helping the vast majority of people with this devastating disease.”
Kalydeco is the first drug available that treats the underlying cause of CF in individuals with specific mutations in the CFTR gene. A patient’s story, who is taking the drug, was highlighted during President Obama’s State of the Union address as a testament of the promise of precision medicine, an approach to treatment that personalizes care and takes into account patients’ individual characteristics.
Source: Vertex Pharmaceuticals Incorporated
Last updated: 3/18/15; 12:10pm EST