Koselugo Approved for NF1 Plexiform Neurofibromas in Younger Patients

The Food and Drug Administration (FDA) has approved Koselugo (selumetinib) oral granules and capsules for pediatric patients aged 1 year and older with neurofibromatosis type 1 (NF1) who have symptomatic, inoperable plexiform neurofibromas (PN). 

Previously Koselugo, an oral selective MEK inhibitor, had only been available in a capsule formulation for patients aged 2 years and older. The treatment was approved in 2020 based on data from the phase 2 SPRINT Stratum I study (ClinicalTrials.gov Identifier: NCT01362803), which included 50 patients with NF1-related symptomatic, inoperable PN. 

The approval of the oral granules was supported by a bioavailability study in healthy adults, as well as exposure matching between patients in the SPRINT Stratum I trial, which evaluated the capsule in patients aged 2 years and older, and the SPRINKLE study (ClinicalTrials.gov Identifier: NCT05309668), which is currently assessing the oral granules in patients 1 year and older. 

Findings showed comparable exposure between the 2 formulations, which supported the extrapolation of efficacy to younger patients. 

The new oral granules formulation of Koselugo is supplied in 5mg and 7.5mg dosage strengths. The oral granules are contained within capsules that should be opened and administered to the patient, within 30 minutes of preparation, in smooth yogurt or fruit puree (eg, apple, banana, pear, or strawberry); the capsule shells should not be swallowed. The oral granules should not be added to liquids or mixed in grapefruit or any juice, puree or jam containing Seville orange. 

The recommended dose for either capsules or oral granules is based on body surface area. Dosage modifications are recommended for patients with hepatic impairment and those on concomitant strong or moderate CYP3A inhibitors or fluconazole. 

This article originally appeared on MPR