FDA approves ivacaftor to treat infants with cystic fibrosis as early as 4 months of age

The FDA has approved ivacaftor for the treatment of cystic fibrosis in infants as young as 4 months old with at least one mutation in their cystic fibrosis transmembrane conductance regulator gene that is responsive to ivacaftor.

Ivacaftor (Kalydeco, Vertex Pharmaceuticals) is already FDA approved to treat cystic fibrosis in patients aged 6 months and older.

Th new approval was based on data from ARRIVAL, a phase 3, open-label, safety cohort study of six children with cystic fibrosis aged 4 months to younger than 6 months. All children had at least one of 10 mutations in the CFTR gene. In this cohort, treatment with ivacaftor demonstrated a similar safety profile to older children and adults.

“Initiating therapy that treats the underlying cause of cystic fibrosis as early as 4 months of age may have the potential to modify the course of the disease,” Margaret Rosenfield, MD, MPH, of the Seattle Children’s Research Institute and department of pediatrics at the University of Washington School of Medicine, said in the release.

“Since the initial approval of Kalydeco more than 8 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,” Reshma Kewalramani, MD, CEO and president of Vertex Pharmaceuticals, stated in the release. “[This] approval is a testament to our relentless efforts, alongside the clinical and scientific community, to reach all people with [cystic fibrosis] who may benefit from our medicines.”