FDA approves selumetinib for pediatric patients 1 year of age and older with neurofibromatosis type 1 with symptomatic, inoperable plexiform neurofibromas
Regulatory approval: On Sept 10, 2025, the FDA approved selumetinib (KOSELUGO; AstraZeneca) granules and capsules for pediatric patients aged ≥1 year with neurofibromatosis type 1 and symptomatic, inoperable plexiform neurofibromas. Selumetinib capsules were previously approved for patients aged ≥2 years. The expanded age indication was supported by bridging data between formulations (relative bioavailability in healthy adults) and exposure matching across pediatric studies, with no new safety signals reported.
FDA approval (Sept 10, 2025)
The FDA approved selumetinib (KOSELUGO; AstraZeneca Pharmaceuticals LP) granules and capsules for pediatric patients aged 1 year and older with neurofibromatosis type 1 (NF1) who have symptomatic, inoperable plexiform neurofibromas (PN). Selumetinib capsules were previously approved for pediatric patients aged 2 years and older with the same condition.
Evidence supporting the expanded age indication
The approval was based on:
- Bridging between the oral granule and the approved capsule formulations using a relative bioavailability study in healthy adults (Study 89).
- Exposure matching between pediatric patients in SPRINT Phase II Stratum I (capsule formulation; ≥2 years) and SPRINKLE (oral granule formulation; ≥1 year).
Similar exposure between formulations was cited as supporting extrapolation of efficacy from patients aged ≥2 years to those aged ≥1 year.
Label highlights
Warnings/precautions include cardiomyopathy, ocular toxicity, gastrointestinal toxicity, skin toxicity, increased creatine phosphokinase, increased vitamin E levels and increased bleeding risk (capsules), and embryo-fetal toxicity. Incidences were updated with data from a larger pediatric population; no new safety signals were identified.
The recommended dose is 25 mg/m² orally twice daily (based on body surface area) until disease progression or unacceptable toxicity.
Full prescribing information is expected to be posted on Drugs@FDA.