FDA Approval Offers New Path for Pediatric NF1 Care

On the 10th of September, the FDA approved KOSELUGO (selumetinib) granules & capsules for children 1 year or older who suffer from neurofibromatosis type 1 (NF1) and who have symptomatic, inoperable plexiform neurofibromas (PN). Until now, approval was limited to children 2 years and older using the capsule form.

This extended approval was built on a bioavailability study in healthy adults comparing the granule and capsule formulations, along with data showing consistent drug exposure between older children treated in the SPRINT Phase II trial (capsules, ≥2 years) and younger children in the SPRINKLE trial (granules, ≥1 year). The findings confirmed that both formulations achieve similar efficacy.

The restructured prescribing information includes safety data from a larger pediatric population, confirming previously known warnings and precautions such as cardiomyopathy, ocular effects, gastrointestinal and skin toxicities, elevated creatine phosphokinase, vitamin E–related risks, and embryo-fetal toxicity. Importantly, no new safety signals were observed.

KOSELUGO has been granted breakthrough therapy and orphan drug status, reflecting its role in addressing a significant unmet need for children living with NF1.

Learn more:  FDA approves selumetinib for pediatric patients 1 year of age and older with neurofibromatosis type 1 with symptomatic, inoperable plexiform neurofibromas