Kalydeco Receives Expanded Indication for Pediatric Patients

September 28, 2020 – The U.S. FDA has approved an expanded indication for Kalydeco® (ivacaftor), manufactured by Vertex Pharmaceuticals. The drug is now indicated to treat patients diagnosed with cystic fibrosis (CF) who are at least four months of age and have one mutation in the CFTR gene that is responsive to ivacaftor based on clinical and/or in vitro assay data. Previously, Kalydeco was approved to treat patients six months of age and up.

Cystic fibrosis is a progressive genetic disease that affects the body’s mucus and sweat glands. Mutations of the cystic fibrosis transmembrane conductance regulator (CFTR) gene cause problems with CFTR protein. Without properly functioning CFTR protein, the body produces abnormally thick, sticky mucus. The mucus builds up in various organs, leading to issues that include inflammation, infection, scarring, malnutrition, and respiratory problems.

In a clinical study, Kalydeco demonstrated a similar safety profile in children aged four months to less than six months as that seen in older children and adults. The drug is the first in its class to receive FDA approval to treat CF in children four months to less than six months of age. Recommended dosing for patients in this age group is one 25mg packet of oral granules mixed with 5mL of soft food or liquid and administered orally every 12 hours with fat-containing food. Patients should weigh at least 5kg (11 pounds).

Kalydeco first received FDA approval in 2012 and is available in both tablet and oral granule dosage forms.