FDA Approves Selumetinib Granules and Capsules for Pediatric Patients With NF1-Associated Symptomatic and Inoperable Plexiform Neurofibromas
On September 10th, 2025, the US Food and Drug Administration (FDA) approved selumetinib granules and capsules for pediatric patients ≥ 1 year of age with neurofibromatosis type 1 (NF1) who have symptomatic and inoperable plexiform neurofibromas (PN).
This approval was based on a relative bioavailability study in healthy adults and exposure matching between patients enrolled in the phase 2 SPRINT Stratum I study assessing the capsule formulation in patients ≥ 2 years of age and the SPRINKLE study assessing the granule formulation in patients ≥ 1 year of age. Similar exposure was demonstrated between the formulations supporting efficacy in these patient populations.
The recommended dose of selumetinib is 25 mg/m2 twice daily until disease progression or unacceptable toxicity, based on body surface area. Prescribing information includes warnings and precautions for cardiomyopathy, ocular toxicity, gastrointestinal toxicity, skin toxicity, increased creatine phosphokinase, increased vitamin E, increased bleeding risk, and embryo-fetal toxicity.
The FDA has previously approved selumetinib for pediatric patients ≥ 2 years of age with NF1 who have symptomatic and inoperable PN.
Source:
FDA approves selumetinib for pediatric patients 1 year of age and older with neurofibromatosis type 1 with symptomatic, inoperable plexiform neurofibromas. Accessed on September 10, 2025. https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-selumetinib-pediatric-patients-1-year-age-and-older-neurofibromatosis-type-1
