Lutetium Lu 177 Dotatate Gains FDA Approval for Pediatric Patients With SSTR-Positive Gastroenteropancreatic Neuroendocrine Tumors

On April 23, 2024, the US Food and Drug Administration (FDA) approved lutetium Lu 177 dotatate for pediatric patients aged 12 and older with somatostatin receptor (SSTR)-positive gastroenteropancreatic neuroendocrine tumors (GEP-NETs) including foregut, midgut, and hindgut neuroendocrine tumors. In January 2018, lutetium Lu 177 dotate was approved for this indication among adult patients.

This approval was based on pharmacokinetic, dosimetry, and safety results from the ongoing, open-label, single-arm NETTER-P study. In this study, a total of 9 adolescent patients with locally advanced/inoperable or metastatic SSTR-positive GEP-NETs (n = 4) or pheochromocytoma/paraganglioma (n = 5) received 7.4 GBq of lutetium Lu 177 dotatate every 8 weeks for a total of 4 doses. Efficacy outcomes from the open-label, active-controlled, NETTER-1 study also helped to determine this approval. In the NETTER-1 trial, adult patients with locally advanced/inoperable or metastatic SSTR-positive midgut carcinoid tumors received lutetium Lu 177 dotatate. The major efficacy end points included absorbed radiation doses in target organs and incidence of adverse reactions after the first treatment cycle. An additional efficacy end point was short-term adverse reactions following treatment. 

At analysis, the adverse event profile was similar to that observed in adult patients. The recommended dose of lutetium Lu 177 dotatate is 7.4 GBq every 8 weeks (± 1 week) for a total of 4 doses. Premedications and concomitant medications should be administered as recommended. A post-marketing requirement was issued to assess the long-term safety of lutetium Lu 177 dotatate in adolescents. 

This is the first FDA approval of a radioactive drug or radiopharmaceutical for pediatric patients with SSTR-positive GEP-NETs. 

Source: 

FDA approves lutetium Lu 177 dotatate for pediatric patients 12 years and older with GEP-NETS. Published online: April 23, 2024. https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-lutetium-lu-177-dotatate-pediatric-patients-12-years-and-older-gep-nets