FDA approves selumetinib for pediatric patients 1 year of age and older with NF1 with symptomatic, inoperable plexiform neurofibromas

Author: Food and Drug Administration

Published On: 11/05/25

Full prescribing information for KOSELUGO will be posted on Drugs@FDA.

The approval was based on adequate bridging between the oral granule and approved capsule formulations in a relative bioavailability (BA) study in healthy adults (Study 89) and exposure matching between the pediatric patient populations in the SPRINT Phase II Stratum I study (capsule formulation, ≥ 2 years of age) and the SPRINKLE study (oral granule formulation, ≥ 1 year of age). Similar exposure between the formulations supports extrapolation of efficacy from pediatric patients ≥ 2 years of age to ≥ 1 year of age.

The selumetinib prescribing information includes warnings and precautions for cardiomyopathy, ocular toxicity, gastrointestinal toxicity, skin toxicity, increased creatine phosphokinase, increased levels of vitamin E and increased bleeding risk (KOSELUGO capsules), and embryo-fetal toxicity. The incidences of warnings and precautions were updated to include data from a larger number of pediatric patients; no new safety signals were identified.

The recommended selumetinib dose, based on body surface area, is 25 mg/m2 orally twice daily, until disease progression or unacceptable toxicity.

Read More

Find upcoming NF Events            Donate to NF          Stay iNFormed