On September 10, 2025, the Food and Drug Administration approved selumetinib granules and capsules for paediatric patients 1 year of age and older with neurofibromatosis type 1 (NF1) who have symptomatic, inoperable plexiform neurofibromas (PN).

FDA previously approved selumetinib capsules for paediatric patients 2 years of age and older with NF1 who have symptomatic, inoperable PN.

Full prescribing information for selumetinib granules and capsules 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 paediatric 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 paediatric 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 (selumetinib capsules), and embryo-fetal toxicity.

The incidences of warnings and precautions were updated to include data from a larger number of paediatric 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.

Selumetinib received breakthrough therapy and orphan drug designations.

FDA expedited programs are described in the Guidance for Industry: Expedited Programs for Serious Conditions-Drugs and Biologics.

Source: FDA