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

April 23, 2024

The following is a message from the Director of the FDA Oncology Center of Excellence, Dr. Richard Pazdur:

On April 23, 2024, the Food and Drug Administration approved lutetium Lu 177 dotatate (Lutathera, Advanced Accelerator Applications USA, Inc., a Novartis company) for pediatric patients 12 years and older with somatostatin receptor (SSTR)-positive gastroenteropancreatic neuroendocrine tumors (GEP-NETs), including foregut, midgut, and hindgut neuroendocrine tumors. Lutetium Lu 177 dotatate received approval for this indication for adults in 2018. 

This represents the first FDA approval of a radioactive drug, or radiopharmaceutical, for pediatric patients 12 years of age and older with SSTR-positive GEP-NETs.

Full prescribing information for Lutathera will be posted here.

Approval was based on pharmacokinetic (PK), dosimetry, and safety data from NETTER-P (NCT04711135), an ongoing, international, multi-center, 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 (PPGL). 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 9 pediatric patients in NETTER-P, including 4 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 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.

NETTER-P was conducted as part of a pediatric Written Request (WR) under the Best Pharmaceuticals for Children Act (BPCA). This 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 1-800-FDA-1088.

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

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