FDA Expands Selumetinib Approval to Younger Pediatric Patients with NF1
The Food and Drug Administration (FDA) announced the expanded approval of Koselugo (selumetinib; AstraZeneca, Wilmington, DE) to include pediatric patients aged 1 year and older with neurofibromatosis type 1 (NF1) and symptomatic, inoperable plexiform neurofibromas (PN). The therapy was initially approved in 2020 only for children aged 2 years and older. Following the new decision from the FDA, Koselugo is approved for oral use in a capsule or granule form.
The expanded indication was supported by data from bridging studies comparing the capsule and oral granule formulations. A phase 2 study (NCT02407405) in adults demonstrated equivalent bioavailability between the 2 formulations. Additionally, exposure matching between the SPRINT phase 2 Stratum 1 study (NCT01362803)—which assessed capsule treatment in participants aged ≥2 years—and the SPRINKLE phase 1/2 study (NCT05309668)—which assessed granule treatment of participants aged ≥1 year—confirmed similar drug exposure in younger children. These findings enabled researchers to extrapolate efficacy data from older pediatric populations to children aged ≥1 year.
The recommended dose of Koselugo is 25 mg/m² orally twice daily, continued until progression or unacceptable toxicity. In a statement from the FDA, the Agency notes that the prescribing information for Koselugo includes warnings and precautions for cardiomyopathy, ocular toxicity, gastrointestinal toxicity, skin toxicity, increased creatine phosphokinase, and embryo-fetal toxicity. Additionally, the capsule formulation includes a warning and precaution for increased levels of vitamin E and increased bleeding risk.
Source: Rddad Y. FDA expands selumetinib use to younger children with NF1 tumors. OncologyNewsCentral.com. Published 10 September, 2025. Accessed 11 September, 2025.
U.S. Food & Drug Administration. FDA approves selumetinib for pediatric patients 1 year of age and older with neurofibromatosis type 1 with symptomatic, inoperable plexiform neurofibromas. Fda.gov. Published 10 September, 2025. Accessed 11 September, 2025.