The US FDA has approved Kalydeco (ivacaftor, Vertex Pharma) as a therapy for cystic fibrosis in infants as young as 4 months old to less than 6 months old with at least one mutation in their cystic fibrosis transmembrane conductance regulator gene.
Kalydeco was already approved in the US and EU for the treatment of CF in patients ages six months and older.
“Since the initial approval of Kalydeco more than eight years ago, we have continued to advance our clinical development program with the goal of treating the underlying cause of cystic fibrosis as early in life as possible,” said Reshma Kewalramani, MD, Chief Executive Officer and President, Vertex. “Today’s approval is a testament to our relentless efforts, alongside the clinical and scientific community, to reach all people with CF who may benefit from our medicines.”
This FDA approval is based on data from a cohort in the 24-week Phase 3 open-label safety cohort (ARRIVAL) consisting of 6 children with CF ages four months to less than six months who have one of 10 mutations in the CFTR gene (G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, G1349D or R117H). This cohort demonstrated a safety profile similar to that observed in older children and adults.
“Initiating therapy that treats the underlying cause of cystic fibrosis as early as four months of age may have the potential to modify the course of the disease,” said Margaret Rosenfeld, MD, MPH, Seattle Children’s Research Institute and Department of Pediatrics, University of Washington School of Medicine.
Kalydeco was first approved in 2012 in the US and is now available in more than 40 countries