FDA Approves Lutetium Lu 177 Dotatate for Pediatric Patients Aged 12 and Older With GEP-NETS

April 24, 2024

On April 23, 2024, the U.S. Food and Drug Administration (FDA) approved (https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-lutetium-lu-177-dotatate-pediatric-patients-12-years-and-older-gep-nets) 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 (https://wayback.archive-it.org/7993/20201222065726/https:/www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-lutetium-lu-177-dotatate-treatment-gep-nets) 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 (https://www.accessdata.fda.gov/drugsatfda_docs/label/2024/208700s031lbl.pdf).

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 (https://www.fda.gov/regulatory-information/search-fda-guidance-documents/expedited-programs-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 (https://www.accessdata.fda.gov/scripts/medwatch/index.cfm) 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 (https://www.fda.gov/about-fda/oncology-center-excellence/project-facilitate) at 240-402-0004 or email OncProjectFacilitate@fda.hhs.gov (mailto:OncProjectFacilitate@fda.hhs.gov).

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