The safety and tolerability of KALYDECO were evaluated in 36 children age 4 months to less than 2 years old with cystic fibrosis (CF). All the children in this study took KALYDECO.
One part of the study evaluated 6 children age 4 months to less than 6 months old.
Mutations eligible to enroll in this study were:
G1244E, G1349D, G178R, G551D, G551S, R117H, S1251N, S1255P, S549N, S549R.
How KALYDECO was given
All children age 4 months to less than 6 months of age received 25 mg of KALYDECO oral granules.
In the clinical study, instruction was provided to give the dose of KALYDECO oral granules mixed into 1 teaspoon of an age-appropriate soft food or liquid, either orally with a syringe or with a spoon, every 12 hours along with a fat-containing food.
Direction was given to ensure the full contents of the syringe were administered.
KALYDECO granules should not be given with a bottle.
Other study results
KALYDECO was approved in people age 4 months to less than 6 months old based on the efficacy shown in studies of KALYDECO in older people as well as the safety assessment in this study.
Talk to your healthcare provider to learn more about how KALYDECO was approved in this age group.