Lutathera Approved for Children With Gastroenteropancreatic Neuroendocrine Tumors

The Food and Drug Administration (FDA) has expanded the approval of Lutathera (lutetium Lu 177 dotatate), a radiolabeled somatostatin analog, to include pediatric patients 12 years of age and older with somatostatin receptor-positive gastroenteropancreatic neuroendocrine tumors (GEP-NETs), including foregut, midgut, and hindgut neuroendocrine tumors. Previously, the treatment was only approved for adults.

The expanded approval was based on pharmacokinetic, dosimetry, and safety data from the ongoing multicenter, open-label, single-arm phase 2 NETTER-P study (ClinicalTrials.gov Identifier: NCT04711135), which included 9 pediatric patients, of which 4 had GEP-NETs. The major outcome measures were absorbed radiation doses in target organs and incidence of adverse reactions after the first treatment cycle. 

Findings showed the safety profile of lutetium Lu 177 dotatate in pediatric patients 12 to 17 years of age was consistent with that observed in adult patients. Comparable results were also observed with regard to the estimated radiation absorbed dose, which was found to be within established organ thresholds for external beam radiation.

The efficacy of lutetium Lu 177 dotatate in pediatric patients with GEP-NETs was extrapolated from the phase 3 NETTER-1 study (ClinicalTrials.gov Identifier: NCT01578239), which supported the original approval in adult patients.

“While GEP-NETs in children and adolescents are rare, the impact can be devastating,” said Dr Theodore Laetsch, trial investigator and Director, Developmental Therapeutics Program, Children’s Hospital of Philadelphia (CHOP), a NETTER-P clinical trial site. “The introduction of radioligand therapy significantly advanced how we treat GEP-NETs, and I’m encouraged that younger patients now have the potential to benefit from this innovation.”