Kalydeco (ivacaftor) was shown to be safe and well-tolerated in 1- and 2-year-old children with cystic fibrosis (CF), based on data from a Phase 3 trial, Vertex Pharmaceuticals announced. Additionally, trial data show that Kalydeco improved sweat chloride levels, a hallmark symptom of CF, in the children under study.
Based on the positive interim results, Vertex said that in the first quarter of 2018, it would seek regulatory approval in the United States and Europe for the use of Kalydeco for treatment of CF in children ages 1 to 2.
“We know that cystic fibrosis is a progressive disease with organ damage already present at birth, so the earlier patients can begin treatment, the greater their potential for improved outcomes,” Jane Davies, MD, from the Royal Brompton Hospital and Imperial College London, and co-lead investigator of the ARRIVAL study, said in a press release.
“These results are incredibly exciting: they suggest that we can begin treating the underlying cause of cystic fibrosis with Kalydeco in children as young as one year of age,” Davies added.
Kalydeco is currently approved by the U.S. Food and Drug Administration (FDA) for the treatment of CF in children ages 2 and older who have one of 38 Kalydeco-responsive mutations in the CFTR gene.
The ongoing, open-label, Phase 3 ARRIVAL study (NCT02725567) is assessing the safety, stability, and effectiveness of Kalydeco in children younger than 2 who have specific mutations affecting the CFTR gene: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, G1349D, or R117H.
The most recent trial data were collected from 18 children who completed 24 weeks of treatment with Kalydeco. Seventeen of these CF patients were enrolled in a rollover study to continue receiving the treatment. The only patient not included in the rollover study was older than 2 and initiated treatment.
Interim data showed that Kalydeco is overall well-tolerated by this group of young children. Similar to earlier findings, the most common adverse events were cough, fever, elevated liver enzymes, and runny nose.
An evaluation of sweat chloride levels showed that after 24 weeks of treatment, the patients showed a mean absolute decrease of 73.5 mmol/L. This meant that median sweat chloride levels had returned to normal.
The treatment also improved pancreatic function, one of the key clinical manifestations of CF.
“We have a significant body of evidence demonstrating Kalydeco’s immediate and long-term benefits and its potential to modify the course of CF,” said Jeffrey Chodakewitz, MD, executive vice president and chief medical officer at Vertex. “These results are an important step in our goal of treating children as early as possible to intervene in this progressive disease.”
The study is ongoing in infants younger than 1.
Vertex plans to present complete ARRIVAL study data at an upcoming medical conference.