Lutathera® Approved by FDA as First Medication for Pediatric Patients with Certain NETs

The U.S. Food and Drug Administration approved Lutathera® as the first therapy specifically for patients 12 to 17 years of age who have somatostatin receptor-positive (SSTR+) gastroenteropancreatic neuroendocrine tumors (GEP-NETs), including foregut, midgut, and hindgut neuroendocrine tumors.

This marks the first time the FDA has approved a radiopharmaceutical for adolescents with SSTR-positive GEP-NETs.

Developed by Novartis, Lutathera (lutetium Lu 177 dotatate) is a peptide receptor radionuclide therapy (PRRT) that selectively targets the somatostatin receptor found on NET cells and delivers radiation specifically to the cancer cells. PRRT aims to harness the power of radioactive atoms to deliver targeted treatment to cells anywhere in the body.

Based on the results of the NETTER-1 trial, the FDA approved Lutathera in January 2018 to treat adults with SSTR-positive GEP-NETs.

The FDA’s approval in April of Lutathera for use in adolescents was based on the NETTER-P trial, a Phase 2 study that launched in August 2022 to evaluate the therapy in patients aged 12-17 years old who have SSTR+ GEP-NETs or pheochromocytomas/paragangliomas. The study is being conducted at several sites in the United States and Europe.

The NETTER-P study reported a safety profile consistent with the adult population studied in NETTER-1, the pivotal trial for approval of Lutathera in adults. The study also found that the estimated absorbed radiation dose in pediatric patients was within established organ thresholds for external beam radiation and comparable to that in adults for the approved dose.

“While GEP-NETs in children and adolescents are rare, the impact can be devastating . . . and the FDA’s approval addresses a critical need for new treatment options for these vulnerable patients,” said Dr. Theodore Laetsch, trial investigator and director of the Developmental Therapeutics Program at The Children’s Hospital of Philadelphia, one of the NETTER-P clinical trial sites.