FDA Approves Selumetinib in Pediatric Neurofibromatosis Type 1

The FDA has approved selumetinib (Koselugo) granules and capsules for the treatment of pediatric patients who are 1 or older with neurofibromatosis type 1 (NF1) and symptomatic, inoperable plexiform neurofibromas (NF1-PNs), according to a release from the FDA.1 Previously, in April 2020, selumetinib capsules were approved for the treatment of pediatric patients 2 years of age and older with NF1-PNs.2 The approval was based on adequate bridging between the oral granule and approved capsule formulations in a relative bioavailability study in healthy adults (Study 89) and exposure matching between the pediatric patient populations in the SPRINT phase 2 Stratum 1 study (NCT01362803) and the SPRINKLE study (NCT05309668). Notably, the similar exposure between formulations supported the extrapolation of efficacy from pediatric patients 2 years and older to patients 1 year and older. The recommended dose of selumetinib, based on body surface area, is 25 mg/m2 orally twice daily until disease progression or unacceptable toxicity. The prescribing information for selumetinib warns about cardiomyopathy, ocular toxicity, gastrointestinal toxicity, and skin toxicity, among other things. References