Vertex Pharmaceuticals Incorporated recently received approval from the United States Food and Drug Administration for Kalydeco® (ivacaftor) to treat children aged between 2 and 5 who suffer with cystic fibrosis (CF) and carry specific mutations in the CFTR gene.
Children who carry one of ten different mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, including G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, S549R and R117H, can now be treated with Kalydeco by Vertex. Cystic fibrosis results from a missing or defective CFTR protein, a consequence of mutations in the CFTR gene.
Before today’s approval, Kalydeco was only approved in America for those older than 6 years old with these specific mutations. It is believed that there are about 300 children in the U.S. between the age of 2 and 5 that carry 1 of these 10 mutations; these 300 children may include 150 with the R117H mutation and 150 with one of the other 9 mutations that cause a gating impairment in the CFTR protein.
In addition, a new oral granule formulation of Kalydeco based on weight (75 mg and 50mg) is now being offered, which can be easily mixed in liquids or soft foods to meet the urgent needs of very young children who may not be capable of swallowing a tablet. The formulation is supported by previously announced outcomes of an open-label Phase 3, 24-week study that assessed both pharmacokinetics and safety of this dosing based on weight of ivacaftor (75 mg or 50 mg, two times per day) in children between 2 and 5 years of age. Now, with this approval, more than 3,400 people are eligible to be treated with Kalydeco across the U.S., Canada, Australia and Europe.
Jeffrey Chodakewitz, Chief Medical Officer and Executive Vice President of the company, stated in a press release: “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. 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.”
Other Recent News About Kalydeco
A new report on cystic fibrosis (CF) in England and Wales further supports evidence of the groundbreaking impact that Kalydeco (ivacaftor) has had on treating CF, and reinforces the Cystic Fibrosis Trust’s urgency for the drug to be made available to the 44 patients with rare CF gene mutations in the two UK nations that currently do not have access to it. Kalydeco is a prescription medicine for the treatment of cystic fibrosis who have the specific G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, G1349D or G970R mutation in the Cystic Fibrosis Transmembrane Regulator (CFTR) gene.