FDA Approves First Radioactive Drug for Children With SSTR-Positive GEP-NETs

Lutetium Lu 177 dotatate marks the first FDA approval of a radioactive drug for some pediatric patients with SSTR-Positive GEP-NETs.

The FDA approved lutetium Lu 177 dotatate (Lutathera) for the treatment of pediatric patients aged 12 years and older with somatostatin receptor (SSTR)-positive gastroenteropancreatic neuroendocrine tumors (GEP-NETs), including hindgut, midgut, and foregut NETs.

This marks the first FDA approval of a radioactive drug, or radiopharmaceutical, to treat pediatric patients 12 years of age and older with SSTR-positive GEP-NETs, according to a release from the agency. Initially, lutetium Lu 177 dotatate received approval from the FDA for this indication in adults in 2018.

The FDA approval was based on dosimetry, pharmacokinetic, and safety data from the NETTER-P trial (NCT04711135). In this ongoing, international, multicenter, open-label, single-arm study, adolescent patients with locally advanced/inoperable or metastatic SSTR-positive GEP-NETs or pheochromocytoma/paraganglioma were treated with lutetium Lu 177 dotatate.

Findings from the NETTER-1 trial (NCT01578239) were also used to determine this drug’s approval, particularly the extrapolation of efficacy outcomes observed in this trial. In this randomized, multicenter, open-label, active-controlled trial, 229 patients with locally advanced/inoperable or metastatic SSTR-positive midgut carcinoid tumors were treated with lutetium Lu 177 dotatate. Of note, findings from this trial supported the original approval of lutetium Lu 177 dotatate in adults.

The safety of lutetium Lu 177 dotatate was assessed in 9 pediatric patients from the NETTER-P trial, which included 4 patients with GEP-NETs. Major outcome measures included the incidence of adverse reactions after the first treatment cycle and absorbed radiation doses in target organs, according to the release. Other outcomes assessed in the trial included short-term adverse reactions after treatment with lutetium Lu 177 dotatate. The adverse reaction profile seen in the NETTER-P trial was similar to those observed in adults.

The FDA recommended dose of lutetium Lu 177 dotatate is 7.4 GBq (200 mCi) every 8 weeks (± 1 week) for a total of 4 doses, according to the agency. Concomitant medications and premedications should be given as recommended. The post-marketing requirement was issued by the FDA to analyze the long-term safety of lutetium Lu 177 dotatate in adolescent patients.

Reference

FDA approves lutetium Lu 177 dotatate for pediatric patients 12 years and older with GEP-NETS. News release. FDA. April 23, 2024. Accessed 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