On April 23, 2024, the U.S. Food and Drug Administration (FDA) approved lutetium Lu 177 dotatate (Lutathera®) for pediatric patients aged 12 and older with somatostatin receptor (SSTR)-positive gastroenteropancreatic neuroendocrine tumors (GEP-NETs), including foregut, midgut, and hindgut neuroendocrine tumors. FDA approved lutetium Lu 177 dotatate for the indication for adults in 2018.

FDA update

It is the first FDA approval of a radiopharmaceutical for pediatric patients aged 12 and older with SSTR-positive GEP-NETs.

Approval was based on pharmacokinetic, dosimetry, and safety data from NETTER-P (NCT04711135), an ongoing, international, multicenter, open-label, single-arm study of lutetium Lu 177 dotatate in adolescent patients with locally advanced, inoperable, or metastatic SSTR-positive GEP-NETs or pheochromocytoma/paraganglioma. Approval was also based on the extrapolation of efficacy outcomes observed in NETTER-1 (NCT01578239), a randomized, multicenter, open-label, active-controlled trial in 229 patients with locally advanced, inoperable, or metastatic SSTR-positive midgut carcinoid tumors, which supported the original approval of lutetium Lu 177 dotatate in adult patients.

Safety was evaluated in nine pediatric patients in NETTER-P, including four patients with GEP-NETs. The major outcome measures were absorbed radiation doses in target organs and incidence of adverse reactions after the first treatment cycle. Additional outcome measures included short-term adverse reactions following treatment with lutetium Lu 177 dotatate. The adverse reaction profile observed in NETTER-P was similar to that observed in adults.

The recommended lutetium Lu 177 dotatate dose is 7.4 GBq (200 mCi) every eight weeks (plus or minus one week) for a total of four doses. Premedications and concomitant medications should be administered as recommended. FDA added a post-marketing requirement to assess the long-term safety of lutetium Lu 177 dotatate in adolescents.

View the full prescribing information for lutetium Lu 177 dotatate.

NETTER-P was conducted as part of a pediatric written request under the Best Pharmaceuticals for Children Act. The application was granted priority review and orphan drug designation; FDA-expedited programs are described in the Guidance for Industry: Expedited Programs for Serious Conditions—Drugs and Biologics.

Healthcare professionals should report all serious adverse events suspected to be associated with the use of any medicine and device to FDA’s MedWatch Reporting System or by calling 800-FDA-1088.

For assistance with single-patient applications for investigational oncology products, healthcare professionals may contact Oncology Center of Excellence’s Project Facilitate at 240-402-0004 or email OncProjectFacilitate@fda.hhs.gov.